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MONDAY                                                                                                                             VOL.19, NO. 50
                       Your Weekly Source for Market and Financial Views
DEC. 12, 2011                                                                                                                        PAGE 1 OF 32
In Geron’s Wake
                                                                                                     Money Raised By Biotech
Deals, Data Spark Stem Cell                                                                             In 2011 Vs. 2010
Interest, But Hurdles Remain                                                                              Jan. 1 – Dec. 9, 2011: $22,777.24M
By Trista Morrison                                                                                        Jan. 1 – Dec. 9, 2010: $18,987.47M
Editor
     SAN DIEGO – Geron Inc.’s exit from the embryonic stem                           11,000
cell field last month shocked many in the biotech industry                            10,000            9,792
– but not the regenerative medicine experts attending the                                                              8,634
                                                                                             9,000
recent 2011 Stem Cell Meeting on the Mesa.
                                                                                                                               7,863
     Some attendees muttered that Geron had never been                                       8,000
able to effectively balance the conflicting risk-reward                                       7,000             6,700
profiles of its stem cell business and its cancer business.




                                                                                  Millions
                                                                                             6,000
Others whispered that the writing had been on the wall
since the departure of longtime CEO Thomas Okarma in                                         5,000                                        4,351   4,425
February. Still others wondered if perhaps the preliminary                                   4,000
data from Geron’s Phase I spinal cord injury trial – the
world’s first of a human embryonic stem cell therapy –                                        3,000
weren’t as good as had been hoped.                                                           2,000
     Geron has maintained the latter isn’t the case – the                                    1,000
company blamed its withdrawal from the stem cell space
on capital scarcity and uncertain economic conditions. (See                                     0
BioWorld Today, Nov. 16, 2011 .)                                                                        Public           Public/           Private
                                                                                                       Of f erings       Other*           Biotechs
     Regardless of the reason, regenerative medicine                          * Includes financings of public biotech firms with the exceptions of
insiders don’t view Geron’s move as an indication that                        public offerings and certain investments from corporate partners.
the stem cell industry is on shaky ground – at least, not
any shakier than it’s ever been. If anything, data and deal-                “has been sorted out,” he said, and advances in research
flow indicate increasing interest in stem cells, although                    tools have improved the consistency of embryonic stem
maintaining that momentum will require creativity on                        cell work as well. On the embryonic side, Geron also blazed
several fronts.                                                             a regulatory trail with the FDA by getting into the clinic.
                                                                                 “The greatest obstacle now is to deliver compelling
Kicking into Gear                                                           clinical proof of concept data,” said Gil Van Bokkelen, CEO of
    Mahendra Rao, director of the National Institutes of                    Athersys Inc. and chairman of the Alliance for Regenerative
Health’s Center for Regenerative Medicine, noted that the                   Medicine. “That’s what investors are looking for; that’s
stem cell field has been significantly derisked over the past
few years. Most of the technical risk with adult stem cells                                                             See Stem Cells, page 4


                                           BIOTECH SHORT SELLING UP SLIGHTLY, BUT NOT ALL BAD ...................... 2
   THIS WEEK IN                            FORM 10 AS IPO ALTERNATIVE? CORONADO WOULD DO IT AGAIN ........ 3
                                           MONEY RAISED BY BIOTECH IN 2011 ................................................... 4
   BIOWORLD                                WEEK IN REVIEW ................................................................................ 5
                                           WORD ON THE STREET, WEEK IN WASHINGTON ..................................... 6

   INSIGHT                                 PHASE I, II & III CLINICAL TRIALS UPDATE: NOVEMBER 2011 .... 7, 11, 15
                                           CONFERENCE DATA ...................................................................... 19-25
                                           NON-U.S. CLINICAL TRIALS DATA; FDA ACTIONS ......................... 26, 28

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                      Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
MONDAY, DEC. 12, 20 1 1                                         BIOWORLD® INSIGHT                                                           PAGE 2 OF 32

A Difference of Opinion                                                            Despite continued volatility in the market, the
Biotech Short Selling Up                                                       measurements of short activity have returned to normal
                                                                               levels.
Slightly, But Not All Bad                                                          There has, however, been a slight widening of the short
By Brian Orelli                                                                interest as a percent of equity over the last two and a half
BioWorld Insight Contributing Writer                                           years between biopharma and Nasdaq stocks, with the
    Short sellers are generally considered blasphemers by                      biopharma stocks closer to 1 .5 percentage points higher
biotech firms. And for good reason; short sellers borrow                        than the Nasdaq stocks, up from the previously mentioned
shares in order to sell them, which puts negative pressure on                  mean of about 1 percentage point. Grimaldi offered up a
the stock. The only way shorts make money is if shares go                      couple of potential explanations: “It may mean that they’re
down.                                                                          betting against the sector or there may just be a larger
    But biotechs don’t have much to complain about, according                  number of companies where it would be profitable to put
to data compiled by BMO Capital Markets. Short sellers are                     on a short position.”
only slightly more active in the biopharma sector than they                        Not every short position is made by an investor who
are for the entire Nasdaq. Looking at the short interest as a                  believes the shares are overvalued. Short sales are used
percent of equity float since the beginning of 2007, the 302                    to hedge long positions in either the individual stock, the
publicly traded biopharmas used in the study had a mean of                     sector, or the entire investor’s portfolio. They can also be
about 6.6 percent of their available shares sold short, which is               used as a tax deferral strategy.
only about a percentage point higher than the short interest                       Shorts also increase liquidity of shares, which can be
for all the Nasdaq stocks.                                                     helpful for biotechs. Higher liquidity, for instance, helps
    Considering the large number of binary events in biotech
– data read-outs, FDA decisions, etc. – Annette Grimaldi,                                                                     See Shorts, page 6
managing director at BMO, thought the absolute difference
might have been larger. One possible reason is that the binary
events also detract short investors because the losses on a
short position are theoretically limitless, unlike a long position
where the most an investor can lose is the initial investment.                                  BioWorld is now
    The means of the short interest ratio – dividing the short
interest position by the average daily trading volume – were
                                                                                                   on Twitter!
nearly identical at 7.8 and 7.6 for Nasdaq and biopharma
stocks, respectively. In other words, it would take almost eight
days for the shorts to cover all their outstanding short sales at
                                                                                               Stay Connected,
the average trading volume.                                                                  Follow Us on Twitter!
    Both the short interest as a percent of equity float and
the short interest ratio spiked in the middle of 2008 during
the peak of the recession. That’s not surprising as both                                   www.twitter.com/bioworld
measurements describe how safe it is to put on a short
position. As the numbers increase, it becomes harder to
unwind the short.

  BIOWORLD® INSIGHT (ISSN 1541-0579) is published every Monday by AHC Media, 3525 Piedmont Road, Building Six, Suite 400, Atlanta, GA 30305.
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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 3 OF 32

Weighing Pros, Cons                                                                 Unlike in an IPO, Coronado didn’t have investment
Form 10 as IPO Alternative?                                                    bankers trotting management around to meet with
                                                                               institutional investors and raise awareness. “With Form 10,
Coronado Would Do It Again                                                     you essentially have to do it yourself,” Sandage explained,
By Trista Morrison                                                             adding that he and chief operating officer Noah Beerman
Editor                                                                         have been “on the road nonstop” talking to investors and
     Four months ago, Coronado Biosciences Inc. embarked on                    analysts, trying to get the word out.
a route to going public not often seen in the biotech industry:                     For now, trading volume among Coronado’s largely
the Form 10 pathway. (See BioWorld Today, July 20, 2011 .)                     retail base is low, but Sandage isn’t overly concerned, noting
     In essence, the Form 10 pathway offers an alternative to the              that “IPOs don’t trade that much after the first week or two
initial public offering (IPO) or reverse merger, seeking to avoid              either.” And although Coronado’s price has fallen, Sandage
the market volatility and high transaction costs of the former                 is encouraged by the fact that the firm was recently picked
as well as the trading restrictions and investor baggage of the                up by Roth Capital Partners with a buy rating and a $27 price
latter. A private firm looking to use the pathway to go public                  target. He’s also optimistic that more analyst coverage and
simply files a Form 10 with the SEC to become a public reporting                interest from institutional investors will come once the firm
company, files an S-1 to register its shares, and applies to trade              transfers to Nasdaq, a process that is already underway.
those shares on a market. (See BioWorld Insight, July 25, 2011 .)                   Beerman also believes Coronado’s stock price
     Coronado got started down the Form 10 road because                        will benefit from the achievement of some near-term
such filings are required for private companies with 500 or                     milestones. The company recently filed an investigational
more stockholders of record. The rule most often affects large                 new drug application for lead program CNDO-201 , pig
private tech firms like Facebook, but Coronado’s angel-backed                   whipworm eggs, and a Phase I trial is underway. Safety
business model made it one of the rare few private biotechs                    data are expected in the first quarter of 2012, and a Phase II
to hit the stockholder threshold. Although a bill is pending in                trial for Chron’s disease is expected to start in the second
the House that may raise that threshold, under current law,                    quarter. Meanwhile Coronado’s second program, which
Coronado had to become a reporting company.                                    involves ex-vivo activation of NK cells, is poised to start a
     Coronado did not, however, have to start publicly                         Phase I/II trial in acute myeloid leukemia early next year.
trading its shares – but the company was interested in                              It remains to be seen whether or not other biotechs
accessing the public markets. President and CEO Bobby                          follow in Coronado’s footsteps and use the Form 10
Sandage said Coronado had considered a reverse merger                          pathway to go public. They may soon have yet another
and even went as far as to identify a suitable shell company                   IPO alternative: a bill that has passed the House and is
to merge into, but new restrictions from the SEC made that                     now in the Senate would raise the Regulation A exemption
option less appealing. Meanwhile, although five biotechs                        to $50 million, providing privately held biotechs with the
have jumped into the IPO queue in the last few months, the                     opportunity to raise money and start trading their shares
window remains far from what most would consider open.                         without filing a Form 10 to become a public reporting
(See BioWorld Today, Nov. 11 , 2011 .)                                         company. (See BioWorld Insight, Nov. 21 , 2011 .) 
     Hence Coronado opted for the Form 10 route. Sandage said
the process is not well understood and “even with experienced
SEC lawyers we were sort of figuring things out as we went
along,” but he believes it was the right decision and would do
it again with future start-ups.
     Coronado debuted on the OTCQB last month at $11 per
                                                                                                ADVERTISE
share. That price was market-driven, determined not by a
team of investment bankers negotiating with institutional
investors, but simply by what a buyer was willing to pay and
                                                                                                  HERE
a seller was willing to accept. Interestingly, Coronado’s price
was higher than that of three-quarters of the biotechs to price                                ...and reach high-level
IPOs this year, even though the Burlington, Mass.-based firm is                         biotechnology professionals every week!
barely in the clinic.
     Since its pricing, however, Coronado’s shares                                           For advertising opportunities in
(OTCQB:CNDO) have dropped 34 percent to $7.25, as of mid                                      BioWorld Insight, please contact
last week. That’s not necessarily unusual for a newly public
biotech – several members of this year’s IPO class have
                                                                                            Stephen Vance at (404) 262-5511
fallen just as hard, although as a group they are down about                                or stephen.vance@ahcmedia.com
8 percent. The drop does, however, illustrate some of the
challenges of taking the Form 10 route.

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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 4 OF 32

                                                                                   Another sign of momentum can be seen in private
   Money Raised By Biotech:                                                    financing for stem cell and regenerative medicine firms.
   Jan. 1 - Dec. 9, 2011                                                       Four such firms have completed private offerings this
                                                                               year, according to data from Roth Capital Partners, and
                                                                               three of them brought in at least $30 million. Additionally,
  19%                                                 43%                      three firms in the field – Argos Therapeutics Inc., TVAX
  ($4,351M)                                           ($9,792M)                Biomedical Inc. and Verastem Inc. – have filed to go public,
                                                                               and Advanced BioHealing was in the initial public offering
                                                                               queue as well before its acquisition.

                                                                               Not Out of the Woods
                                                                                    So is the financial situation for regenerative medicine
                                                                               companies really that much worse than for biotech as a
                                                                               whole, as Geron’s retraction indicated? It’s somewhat hard
                                                                               to believe, sitting in the brand-spanking-new $127 million
                                                                               laboratories of the Sanford Consortium for Regenerative
                                                                               Medicine, listening to so many examples of progress and
                                                                               success. But according to Geoff Whitehead, principal with
                                                                               Flagship Ventures, the answer is yes.
                                                                                    The outlook for stem cell firms is not quite as dire on
  38%               Public Offerings               43% ($9,792M)               the private equity side. Venture capitalists are looking
  ($8,634M)                                                                    for transformative ideas, large markets and compelling
                    Public/Other                   38% ($8,634M)
                                                                               entrepreneurs, so the fact that a lot of stem cell work is early
                    Private Biotechs               19% ($4,351M)               stage and hard to value “doesn’t really factor into things,”
                                                                               Whitehead said. Flagship has three regenerative medicine
                                                                               firms in its portfolio.
                                                                                    But on the other hand, Whitehead noted that “early
Stem Cells                                                                     stage VCs like to invest in pioneers,” and the stem cell field’s
Continued from page 1
                                                                               pioneers don’t have the best track record thus far. He then
what pharma is looking for. Once that data gets delivered, it                  ticked off a dozen reasons why it would be easier for the
will change the mindset in a big way.”                                         average VC to say “no” to a stem cell investment than “yes.”
     In the adult stem cell space, data is starting to trickle                      Stem cell start-ups do have the benefit of alternative
out. Morrie Ruffin, managing director of the Alliance for                       options like grant funding – the California Institute of
Regenerative Medicine, noted that while the media were busy                    Regenerative Medicine (CIRM) has been doling out money
reporting on Geron, many missed the fact that Mesoblast                        for several years now. But Edward Lanphier, president
Ltd.’s allogeneic adult stem cell product Revascor significantly                and CEO of Sangamo BioSciences Inc., noted that the only
reduced cardiovascular deaths in a randomized, placebo-                        companies able to go public these days are in Phase III, and
controlled Phase II trial, and Aastrom Biosciences Inc.’s                      “is CIRM going to take someone to Phase III? I don’t think so.
autologous expanded cell therapy ixmyelocel-T significantly                     There’s a need for venture capital. I’m not optimistic that it’s
reduced treatment failures in a randomized, placebo-                           going to be met, but there’s a need.”
controlled Phase II critical limb ischemia trial. (See BioWorld                     Even if a stem cell company does get public, that
Today, Nov. 16, 2011 .)                                                        doesn’t mean their fortunes will improve. Lisa Walters-
     There’s been good news on the partnering front as well.                   Hoffert, managing director with Roth, presented an analysis
Every panel at the conference paid homage to Mesoblast’s                       showing that just nine public stem cell and regenerative
$350 million stem cell deal with Cephalon Inc. (now part of                    medicine firms have raised money this year – less than $140
Teva Pharmaceutical Industries Ltd.), which carried a whopping                 million altogether, and with a hefty average 67.5 percent
$130 million up front, as well as to Shire plc’s $750 million buy-             warrant coverage. Since their offerings, the firms have seen
out of regenerative medicine firm Advanced BioHealing Inc.                      their shares fall an average of 24 percent, and only one
(See BioWorld Today, May 19, 2011, and Dec. 9, 2010.)                          company – StemCells Inc. – is in the green.
     “Things move forward at a certain rate and then kick into                      Further, despite the oft-mentioned Mesoblast and
gear,” said Robert Preti, president and chief scientific officer                 Advanced BioHealing deals, business development in the
of Progenitor Cell Therapy LLC, which itself was recently the                  space has yet to catch fire.
subject of some business development activity, having been                          “We spend a lot of time talking to pharma,” said
acquired by NeoStem Inc. NeoStem also acquired stem cell firm                   Sangamo’s Lanphier. “As it relates to cell therapy, there is
Amorcyte Inc. earlier this year. (See BioWorld Today, July 18, 2011 .)                                                  See Stem Cells, page 5
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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 5 OF 32


                                                    WEEK               IN       REVIEW
   Financings
   Dendreon Corp. raised $125 million by monetizing its share of royalties on Victrelis (boceprevir).
   EMBL Ventures GmbH closed a $53.5 million fund for German life science companies.
   Forbion Capital Partners closed its second FCF 1 Co-Investment Fund at about $50 million.
   Gilead Sciences Inc. priced $3.7 billion worth of senior unsecured notes in a public offering.

   Deals
   Biogen Idec Inc. entered a $300 million joint venture with Samsung for biosimilars.
   Medicis Pharmaceutical Corp. acquired the assets of Graceway Pharmaceuticals LLC for $455 million.
   Molecular Partners AG signed an immunology discovery deal with Janssen Biotech Inc.
   Momenta Pharmaceuticals Inc. acquired Virdante Pharmaceuticals Inc.’s protein sialylation programs.

   . . . And More
   See BioWorld Today for coverage of the San Antonio Breast Cancer Symposium.
   Affymax Inc.’s peginesatide got a thumbs up from an FDA advisory committee.
   Curemark LLC reported positive Phase III data with CM-AT in children with autism.
   Genentech Inc./Roche AG’s pertuzumab had good Phase III breast cancer data.
   Theratechnologies Inc. is halting its chronic obstructive pulmonary disease muscle wasting program and
   downsizing by 60 percent.
   Unigene Laboratories Inc.’s parathyroid hormone was dropped by partner GlaxoSmithKline plc.


Stem Cells                                                                     “high-risk work” that neither grant-funded researchers nor
Continued from page 4                                                          companies can stomach. Nonprofits should also focus on
                                                                               the translational work that academics aren’t interested in
interest, but there is a higher bar. Whether it’s safety, efficacy,             because it won’t result in a published paper – the “work that
cost of goods analysis . . . you’ve got to paddle your own canoe               is repetitive but essential to derisk the program to a point
a lot longer.”                                                                 where investors or pharma can pick it up,” she said.
     Matthias Steger, global head of research and technology                        Meanwhile, history (and Geron) has proven the
partnering with Roche AG, provided further perspective.                        traditional drug development business model isn’t a
While he refuted the notion that big pharma is not involved                    particularly good fit for stem cell firms. Whether public or
in stem cells, pointing to Roche’s own significant investment,                  private, the firms that have managed to stay in business
he cautioned that big pharma “can just not afford running                      are those that have, more often than not, found alternative
into big failures. We have to sort out the science. It would be a              sources of funding.
mistake to rush. I think we are making progress, but we have                        Sangamo, for example, has outlicensed its zinc finger
to be a little bit patient.”                                                   protein technology in non-core fields like agriculture and
                                                                               research tools. Cytori Therapeutics Inc. is bringing in
Just Keep Paddling                                                             revenue by selling its Celution adult stem cell processing
     The trick will be for stem cell firms to figure out how they                system in Europe, where it has received a CE Mark, while it
can afford to patiently wait for the catalyst that captures big                navigates the U.S. regulatory path. Organovo Inc. uses its
pharma’s attention, or the proof-of-concept data that drive                    platform to create disease models for partners, as well as
public market interest.                                                        in its own drug development. Neostem generates revenues
     The experts at the conference said it will require an                     from a cell therapy manufacturing business, a stem cell
unprecedented level of public-private partnership. “It can’t                   bank and several initiatives in China, which help support
be industry alone, or academics alone, or government alone                     the cost of developing its cell therapy pipeline.
– there must be collaboration,” said NIH’s Rao. He added                            The approaches are unconventional, but Greg Lucier,
that there are far more stem cell trials being conducted by                    chairman and CEO of Life Technologies Corp., thinks
academics than by industry, but the former have “no idea how                   that’s what the regenerative medicine industry needs. “I
to take it to the next level.” That’s where government needs to                do believe this is an area where we should be pushing the
focus its efforts, he said.                                                    frontiers,” he said. “We’re positive, we’re confident and we
     Susan Solomon, CEO of the New York Stem Cell                              are investing in this area. The horizon we see is near term
Foundation, added that nonprofits can play a role by doing the                  and incredibly bright.” 

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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 6 OF 32


                                            WORD                ON        THE           STREET
  “If you’re a great company with great promise, there’s money out there.”
  – Geoff Whitehead, principal with Flagship Ventures, on funding difficulties for stem cell firms

  “The world of pharma is waking up [to the fact] that this is going to be the next frontier.”
  – Greg Lucier, chairman and CEO of Life Technologies Corp., on stem cells

  “You want to have Track 1 , 2, 3, 4 and 5 – if you only have Track 1 and Track 5, no one wants to give it a shot.”
  – William Hicks, partner with Mintz Levin, on the need to have multiple avenues to going public

  “The hurdle rates for companies in development are being set higher than they have been in the past. Having said that,
  we love being in this industry.”
  – Anthony Marucci, president and CEO, Celldex Therapeutics Inc.




                                            WEEK               IN       WASHINGTON
  The FDA’s latest proposed user fee structure for biosimilars will charge 10 percent of the new drug application fee, plus
  a development fee.

  The U.S. Patent and Trademark Office and China’s State Intellectual Property Office will expedite their patent
  examinations, which should allow applicants to obtain corresponding patents faster in each country.


Shorts                                                                             While shorts might be best ignored from an industry
Continued from page 2
                                                                               perspective, Grimaldi thinks individual firms should
firms sell more shares in at-the-market (ATM) offerings, a                      probably consider why investors are shorting the shares.
public offering alternative that has slowly gained traction over               “Especially large short interest is a signal that differs from
the past few years. Inhibitex Inc. raised $20 million through                  what management is saying. For that particular company,
an ATM last month. (See BioWorld Insight, Sept. 7, 2009.)                      they should look to see if there are any underlying messages
    And short sellers can actually have a positive effect on                   in that position.”
shares if a company produces positive news. A short squeeze                        China Biologic Products Inc., Cumberland Pharma-
occurs as the short sellers cover their sales by buying back                   ceuticals Inc., MannKind Corp., BioTime Inc., and Oncolytics
shares, amplifying the upward movement of the stock in                         Biotechnology Inc. currently top the list of companies with
response to the good news.                                                     the highest short interest. 




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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 7 OF 32

                                         FDA Approvals In November
                Company                                       Drug                                               Indication
 Bristol-Myers Squibb Co.                     Erbitux                                   Head and neck cancer
 EUSA Pharma Inc.                             Erwinase                                  Acute lymphoblastic leukemia
 Incyte Corp.                                 Jakafi                                    Myelofibrosis
 IntelGenx Corp.                              CPI-300                                   Major depressive disorder
 Millennium                                   Velcade                                   Updated label for multiple myeloma
 Pacira Pharmaceuticals Inc.                  Exparel                                   Postsurgical pain relief
 Regeneron Pharmaceuticals Inc.               Eylea                                     Wet age-related macular degeneration
 Spectrum Pharmaceuticals Inc.                Zevalin                                   Removed bioscan requirment for non-Hodgkin’s
                                                                                        lymphoma
 Transcept Pharmaceutical Inc.                Intermezzo                                Insomnia

                   Phase I Clinical Trials Update: November 2011
 Company                   Product            Description                 Indication               Status (Date)#
 (Location)
 CANCER
 Bavarian Nordic           CV-301             Off-the-shelf immu- Metastatic breast A trial with 26 heavily pretreated patients
 A/S (Kvistgard,                              notherapy           or ovarian cancer who received monthly vaccinations
 Denmark)                                                                           showed a median time to progression of
                                                                                    2.5 months in the breast cancer group,
                                                                                    and median overall survival was 13.7
                                                                                    months (11/10)
 Celldex           CDX-1127                   A co-stimulatory            Malignant solid          Started a Phase I study (11/9)
 Therapeutics Inc.                            molecule on T cells         tumors or hema-
 (Needham, Mass.)                                                         tologic cancers
 CureVac GmbH              CV9201             mRNA-based can-             Non-small-cell           Phase I/IIa data showed the drug was well
 (Tuebingen,                                  cer vaccine                 lung cancer              tolerated and biologically active (11/8)
 Germany)
 CytRx Corp. (Los          INNO-206           A tumor-targeting           Sarcoma                  Phase Ib/II trial data determined a maxi-
 Angeles)                                     conjugate of doxo-                                   mum tolerated dose of the drug deliver-
                                              rubicin                                              ing doxorubicin at the equivalent of three
                                                                                                   and a half times standard doxorubicin
                                                                                                   doses (11/1)
 Hutchison                 Epitnib            An orally active            Cancer                   Started the first-in-human Phase I trial
 MediPharma Ltd.           (HMPL-813)         small molecule                                       (11/7)
 (London)                                     inhibitor targeting
                                              the epidermal
                                              growth factor
                                              receptor
 Infinity                  IPI-145            Oral inhibitor of           Advanced                 Started two Phase I trials (11/1)
 Pharmaceuticals                              phosphinositide-3-          hematologic
 Inc. (Cambridge,                             kinase delta and            malignancies
 Mass.)                                       gamma
 Lentigen Corp.            LG631              Gene therapy                Cancer                   Started a Phase I trial (11/15)
 (Gaithersburg, Md.)
 Senesco             SNA01-T                  Intravenous infusion Multiple                        Initiated patient dosing in its Phase Ib/IIa
 Technologies Inc.                                                 myeloma                         study (11/4)
 (Bridgewater, N.J.)

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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 8 OF 32
 Company                   Product            Description                 Indication               Status (Date)#
 (Location)
 Silence                   Atu027             siRNA drug                  Solid tumors             Phase I data showed 37% of patients had
 Therapeutics plc                                                                                  stable disease after treatment (11/9)
 (London)
 CARDIOVASCULAR
 Cardiome                  Vernakalant        Oral therapy                Atrial fibrillation      Recently completed an additional multi-
 Pharma Corp.                                                                                      ple rising-dose Phase I study and data
 (Vancouver, British                                                                               showed the drug was well tolerated (11/7)
 Columbia) and
 Merck & Co. Inc.
 (Whitehouse
 Station, N.J.)
 Nile                Cenderitide              Subcutaneous                Chronic heart            Phase I data showed that weight-based
 Therapeutics Inc.                            infusion                    failure                  doses achieved target PK levels when
 (San Mateo, Calif.)                                                                               delivered by subcutaneous pump, and
                                                                                                   that it was well tolerated, bioavailable
                                                                                                   and reduced PK variability compared to a
                                                                                                   fixed-dose regimen (11/17)
 Pozen Inc. (Chapel PA32540                   A combination of            Gastrointestinal         Phase I data showed it was associated
 Hill, N.C.)                                  325 mg aspirin and          risk in patients         with greater platelet inhibition when
                                              40 mg immediate-            who require dual         dosed 10 hours apart from Plavix com-
                                              release omeprazole          antiplatelet ther-       pared to synchronous administration of
                                                                          apy and gastro-          aspirin, clopidogrel and delayed-release
                                                                          protection               omeprazole at day seven (11/15)
 CENTRAL NERVOUS SYSTEM
 Acadia                    AM-831             A small molecule   Schizophrenia                     FDA cleared it to begin a Phase I trial (11/8)
 Pharmaceuticals                              that combines mus-
 Inc. (San Diego)                             carinic m1 partial
                                              agonism with dopa-
                                              mine D2 and sero-
                                              tonin 5-HT2A
                                              antagonism
 Anavex Life               Anavex 2-73        The first of a new          Alzheimer’s              Completed a Phase I single ascending
 Sciences Corp.                               class of oral drugs         disease                  dose trial of Anavex, showing it was well
 (Hoboken, N.J.)                                                                                   tolerated below the 55 mg to 60 mg dose
                                                                                                   with only mild adverse events (11/15)
 Cara              CR845                      A peptide-based             Acute postopera- Started its first Phase I trial of an oral for-
 Therapeutics Inc.                            kappa opioid ago-           tive pain        mulation (11/23)
 (Shelton, Conn.)                             nist
 Neuralstem Inc.           Stem cell          Stem cell therapy           Amyotrophic              Dosed its first patient in the trial (11/28)
 (Rockville, Md.)          therapy            used in the cervical        lateral sclerosis
                                              region of the spine
 NeuroDerm Ltd.            ND0611             Administered sub-           Parkinson’s              Met all primary and secondary endpoints
 (Ness Ziona, Israel)                         cutaneously by a            disease                  in a Phase I/II trial (11/10)
                                              dermal patch
 Probiodrug AG             PQ912              A glutaminyl                Alzheimer’s              Phase I data demonstrated it was safe and
 (Halle, Germany)                             cyclase inhibitor           disease                  well tolerated (11/15)
 Selecta                   SEL-068            A nicotine vaccine          Smoking cessa-           Started a Phase I trial (11/22)
 Biosciences Inc.                                                         tion and relapse
 (Watertown, Mass.)                                                       prevention


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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 9 OF 32
 Company                   Product            Description                 Indication               Status (Date)#
 (Location)
 DIABETES
 Akebia            AKB-9778                   A human protein             Diabetic macular Started a Phase I trial (11/22)
 Therapeutics Inc.                            tyrosine phospha-           edema and dia-
 (Cincinnati)                                 tase beta inhibitor         betic retinopathy
 Concert                   CTP-499            Controlled-release          Diabetic                 Phase I data showed it was well tolerated
 Pharmaceuticals                              version; analogue           nephropathy              at single doses up to and including 1 ,800
 Inc. (Lexington,                             of 1-([S]-5-                                         mg (11/15)
 Mass.)                                       hydroxyhexyl)-3,7-
                                              dimethylxanthine
 Convergence               CNV2197944         A calcium channel           Chronic pain             Initiated a Phase I trial (11/22)
 Pharmaceuticals                              blocker
 Ltd. (Cambridge,
 UK)
 DARA                      DB959              A peroxisome pro-           Type II diabetes         Phase Ib data showed a safety profile
 BioSciences Inc.                             liferator activated                                  comparable to placebo when given as
 (Raleigh, N.C.)                              receptor-delta/                                      once-daily doses for seven days and was
                                              gamma agonist                                        safe and well tolerated throughout the
                                                                                                   40-fold dose range tested (11/2)
 Isis                      ISIS-GCGRRx        Antisense drugs             Type II diabetes         Started Phase I studies (11/3)
 Pharmaceuticals           and ISIS-
 Inc. (Carlsbad,           GCCRRx
 Calif.)
 PhaseBio                  Glymera            A recombinant glu- Hyperglycemia                     Phase I/IIa data demonstrated statistically
 Pharmaceuticals                              cagon-like peptide-1 in Type II                      significant reductions in fasting glucose
 Inc. (Malvern, Pa.)                          analogue             diabetes                        glycemic load following meal tolerance
                                                                                                   testing and in average daily glucose (11/16)
 INFECTION
 AVIR Green Hills          deltaFLU           Intranasal flu              Influenza                The vaccine was well tolerated and signif-
 Biotechnology                                vaccine                                              icantly increased influenza-specific anti-
 AG (Vienna,                                                                                       bodies in a Phase I/II trial (11/8)
 Austria)
 Inhibitex Inc.            INHX-189           Monotherapy;                Hepatitis C virus        Data showed INX-189 dosed at 100 mg
 (Atlanta)                                    nucleotide poly-                                     once daily for seven days in combination
                                              merase inhibitor                                     with ribavirin resulted in a median HCV
                                                                                                   RNA reduction from baseline of -0.379
                                                                                                   log10 (11/30)
 Inovio                    SynCon             Avian influenza             Avian flu                A single intradermal electroporation
 Pharmaceuticals                              vaccine                                              boost of its vaccine generated hemagglu-
 Inc. (Blue Bell, Pa.)                                                                             tination inhibition titers against six differ-
                                                                                                   ent unmatched strains of H5N1 and it
                                                                                                   generated a fourfold or greater rise in HAI
                                                                                                   titers in 50% of boosted subjects in a
                                                                                                   Phase I study (11/18)
 Novacta                   NVB302             An oral treatment           Hospital-                Started dosing the first healthy volun-
 Biosystems Ltd.                                                          acquired                 teers in a Phase I trial (11/3)
 (London)                                                                 Clostridium
                                                                          difficile
                                                                          infections



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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 10 OF 32
 Company                   Product            Description                 Indication               Status (Date)#
 (Location)
 MISCELLANEOUS
 Aeolus                    AEOL 10150         A broad-spectrum            Acute radiation          Pilot study results of AEOL 10150 and
 Pharmaceuticals                              catalytic antioxi-          syndrome                 G-CSF drug Neupogen confirmed that it
 Inc. (Mission Viejo,                         dant designed to                                     does not interefer with the positive
 Calif.)                                      neutralize reactive                                  effects of Neupogen on the hematopoiet-
                                              oxygen and nitro-                                    ic syndrome (11/2)
                                              gen species
 Akebia            AKB-6548                   Oral hypoxia-               Anemia associat-         Phase Ib/IIa data showed it was well toler-
 Therapeutics Inc.                            inducible factor-           ed with chronic          ated and enhanced erythropoiesis (11/16)
 (Cincinnati)                                 prolyl hydroxylase          kidney disease
                                              inhibitor
 AlloCure Inc.             AC607              A bone marrow-              Acute kidney             Phase I data showed it was safe and well
 (Burlington, Mass.)                          derived mesenchy-           injury                   tolerated, and treated subjects experi-
                                              mal stem cell                                        enced a lower incidence of AKI, reduced
                                              therapy                                              length of hospital stay and reduced hos-
                                                                                                   pital readmission rates compared to a
                                                                                                   cohort of historical controls (11/14)
 Alnylam                   ALN-TTR01          An RNAi therapeu-           TTR-mediated             Phase I data showed statistically signifi-
 Pharmaceuticals                              tic targeting trans-        amyloidosis              cant reductions in serum TTR protein lev-
 Inc. (Cambridge,                             thyretin                                             els in ATTR patients (11/22)
 Mass.)
 Bellicum                  CaspCIDe           Technology used to Graft-vs.-host                    Results of the first clinical study showed
 Pharmaceuticals                              eliminate donor T  disease                           it worked quickly and effectively without
 Inc. (Houston)                               cells                                                compromising the function of the cell
                                                                                                   (11/4)
 Chimerix Inc.             CMX001             A lipid-antiviral-    Kidney disease                 Phase I/II data showed it was generally
 (Durham, N.C.)                               conjugate designed                                   safe and well tolerated when adminis-
                                              to deliver cidofovir-                                tered to renal transplant and hematopoi-
                                              diphosphate                                          etic stem cell transplant patients (11/14)
 Geron Inc. (Menlo         hESC therapy Human embryonic                   Acute spinal             Company is stopping recruitment to its
 Park, Calif.)                          stem cell therapy                 injury                   Phase I trial due to economic conditions
                                                                                                   (11/16)
 Pluristem         PLX-PAD                    Cells derived from          Critical limb            Phase I data showed that it met all the
 Therapeutics Inc.                            the firm’s PLX              ischemia                 endpoints, demonstrating a safe immuno-
 (Haifa, Israel)                              (PLacental eXpand-                                   logic profile at all dosage levels and
                                              ed) platform                                         showing potential efficacy (11/4)
 Provesica Ltd.            XEN-D0501          An antagonist of            Overactive               Phase I data showed it was safe and well
 (Cambridge, UK)                              TRPV1                       bladder                  tolerated (11/28)
 Notes:
 Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.
 # The date indicated refers to the BioWorld Today issue in which the news item can be found.




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MONDAY, DEC. 12, 20 1 1                                         BIOWORLD® INSIGHT                                                            PAGE 11 OF 32

                   Phase II Clinical Trials Update: November 2011
 Company                   Product              Description                 Indication            Status (Date)#
 (Location)
 AUTOIMMUNE
 Adeona           Trimesta                      Oral estriol candi-         Multiple              It will be tested in a Phase II trial evaluating
 Pharmaceuticals                                date                        sclerosis             its potential effect on cognitive dysfunc-
 Inc. (Ann Arbor,                                                                                 tion in female MS patients (11/14)
 Mich.)
 Avanir             AVP-923                     An NMDA antago-             Neuropathic           Enrolled the first patient in a Phase II trial
 Pharmaceuticals                                nist and sigma-1            pain in multi-        expected to enroll 400 patients worldwide
 Inc. (Aliso Viejo,                             agonist                     ple sclerosis         (11/4)
 Calif.)
 Galapagos NV              GLPG0634             JAK1 inhibitor              Rheumatoid            Achieved the primary endpoint of signifi-
 (Mechelen,                                                                 arthritis             cant improvement in the signs and symp-
 Belgium)                                                                                         toms of RA in a Phase IIa trial (11/28)
 KAI             KAI-4169                       Long-acting intrave- Secondary                    Phase II data showed it was effective, with
 Pharmaceuticals                                nous calcium sens-   hyperparathy-                33% and 49% reductions in parathyroid
 Inc. (South San                                ing receptor agonist roidism                      hormone (PTH) from baseline in the 5-mg
 Francisco)                                                                                       and 10-mg dose groups, respectively (11/14)
 Opexa                     Tovaxin              A T-cell therapy            Relapsing-            Phase IIb data showed it was well tolerated,
 Therapeutics                                                               remitting mul-        with no serious adverse events, and dem-
 Inc. (The                                                                  tiple sclerosis       onstrated encouraging results in the reduc-
 Woodlands, Texas)                                                                                tion of the annualized relapse rate and
                                                                                                  improvement in disease progression (11/15)
 SuppreMol                 SM101                A soluble version of Lupus                        Started dosing in a Phase IIa trial (11/29)
 GmbH                                           the Fc gamma recep-
 (Martinsried,                                  tor IIb
 Germany)
 CANCER
 Biothera Inc.             Imprime PGG          An immunomodula-            Non-small-cell        Completed enrollment of 90 patients in its
 (Eagan, Minn.)                                 tor drug                    lung cancer           Phase II trial in combination with Erbitux
                                                                                                  (11/18)
 Celldex                   CDX-110              Rindopepimut                EGFRvIII-             Phase II data showed final overall survival
 Therapeutics                                                               positive glio-        of 24.6 months from diagnosis, compared
 Inc. (Needham,                                                             blastoma              to 15.2 months for a historic control (11/22)
 Mass.)
 Exelixis Inc.             Cabozantinib         MET and VEGFR2              Hormone          Initiated a Phase II trial (11/22)
 (South San                                     inhibitor                   receptor-posi-
 Francisco)                                                                 tive breast can-
                                                                            cer with bone
                                                                            metastases
 Galena                    NeuVax               Vaccine                     Breast cancer         Phase II data showed that it may provide a
 Biopharma Inc.                                                                                   meaningful clinical benefit in patients with
 (Lake Oswego,                                                                                    less aggressive forms of the disease (11/7)
 Ore.)
 Jennerex Inc.             JX-594               Oncolytic virus             Advanced liver Started the Phase IIb trial in patients who
 (San Francisco)                                                            cancer         failed prior therapy with Nexavar (11/7)
 KAEL-GemVax               GV1001               Telomerase peptide          Non-small-cell        Interim Phase II data demonstrated an 80%
 Co. Ltd. (Seoul,                                                           lung cancer           immune response rate (11/16)
 South Korea)


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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 12 OF 32
 Company                  Product              Description                 Indication            Status (Date)#
 (Location)
 Ligand           Melphalan                    Captisol-enabled            Cancer                Phase II data showed that it met the
 Pharmaceuticals                               propylene glycol-                                 requirements for establishing bioequiva-
 Inc. (San Diego)                              free melphalan                                    lence to Alkeran (11/3)
 MediGene AG              EndoTAG-1            A composition of            Hormone-        Phase II trial started (11/9)
 (Martinsried,                                 paclitaxel combined         receptor-
 Germany)                                      with neutral and            positive, HER2-
                                               positive lipids             negative
                                                                           breast cancer
 Peregrine             Bavituximab             A phosphatidylser-          Locally               Company reported a 23.2-month median
 Pharmaceuticals                               ine-targeting mono-         advanced or           overall survival from a single-arm Phase II
 Inc. (Tustin, Calif.)                         clonal antibody             metastatic            trial of bavituximab in combination with
                                                                           breast cancer         carboplatin and paclitaxel, compared with
                                                                                                 a published study showing median OS of 16
                                                                                                 months with carboplatin and paclitaxel
                                                                                                 alone (11/23)
 YM BioSciences           Nimotuzumab Anti-EGFR antibody                   Recurrent dif- Phase II data showed it was well tolerated
 Inc. (Mississauga,                                                        fuse intrinsic when administered to pediatric patients
 Ont.)                                                                     pontine glioma (11/1)
 CARDIOVASCULAR
 Aastrom                  Ixmyelocel-T         A patient-specific    Critical limb               Phase II data showed patients in the treat-
 Biosciences Inc.                              multicellular therapy ischemia                    ment arm had a 62% reduction in risk rela-
 (Ann Arbor, Mich.)                                                                              tive to placebo in the primary endpoint of
                                                                                                 time to first occurrence of treatment failure
                                                                                                 (11/15)
 Mesoblast Ltd.           Revascor             Stem cell treatment         Congestive            Phase II data showed it did not provoke any
 (Melbourne,                                                               heart failure         adverse immune responses and there was
 Australia)                                                                                      a 78% reduction in the rate of serious cardi-
                                                                                                 ac events (11/16)
 Resverlogix              RVX-208              A small molecule            Coronary              Started dosing in its Phase IIb study (11/3);
 Corp. (Calgary,                               designed to stimu-          artery disease        the study is fully enrolled with 176 subjects
 Alberta)                                      late endogenous                                   (11/29)
                                               ApoA-I production
 Sanofi SA (Paris) REGN727/                    A fully human anti-         Elevated low-         Phase II data showed that after 12 weeks of
 and Regeneron     SAR236553                   body targeting pro-         density lipo-         treatment patients achieved mean LDL-C
 Pharmaceuticals                               protein convertase          protein               reductions ranging from about 30% to
 Inc. (Tarrytown,                              subtilisin/kexin            cholesterol           more than 65%, depending on the dosing
 N.Y.)                                         type 9                                            regimen; the control arm had a mean
                                                                                                 reduction of 10% (11/11)
 Theravance Inc.          TD-4208              Inhaled long-acting         Chronic               Phase IIa data showed it met the primary
 (South San                                    muscarinic antago-          obstructive           endpoint, with both study doses demon-
 Francisco)                                    nist                        pulmonary             strating a statistically significant mean
                                                                           disease               change from baseline in peak forced expi-
                                                                                                 ratory volume in one second compared to
                                                                                                 placebo (11/16)
 Trophos SA               TRO40303             A mitochondria pore Cardiac isch-                 Treated the first patient in a Phase II study
 (Marseille, France)                           modulator           emia-reperfu-                 (11/1)
                                                                   sion injury in
                                                                   acute myocar-
                                                                   dial infarction



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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 13 OF 32
 Company                  Product              Description                 Indication            Status (Date)#
 (Location)
 Verona Pharma            RPL554               A mixed phosphodi- Chronic                        Demonstrated bronchodilator effects in a
 plc (London)                                  esterase 3/4 inhibi- obstructive                  Phase IIa trial (11/11)
                                               tor                  pulmonary
                                                                    disease
 CENTRAL NERVOUS SYSTEM
 BioDelivery              BEMA Bupre-          Formulation of              Opioid                Started a confirmatory study, which will
 Sciences                 norphine/            buprenorphine and           dependence            select the final doses to be used in the piv-
 International            Nalaxone             naloxone using                                    otal bioequivalence study compared to
 Inc. (Raleigh, N.C.)                          BDSI’s BioErodible                                Suboxone (11/11)
                                               MucoAdhesive drug
                                               delivery technology
 Clinuvel         Scenesse                     Afamelanotide; pho- Erythropoietic Phase II data showed it appeared to be
 Pharmaceuticals                               toprotective drug   protoporphyria effective in preventing severe pain result-
 Ltd. (Melbourne,                                                                 ing from sun exposure (11/4)
 Australia)
 Cytokinetics Inc. CK-2017357                  A fast skeletal mus-   Amyotrophic                Started a second cohort of an ongoing
 (South San                                    cle troponin activator lateral                    Phase II trial (11/2); opened enrollment in its
 Francisco)                                                           sclerosis                  third Phase II trial (11/30)
 Knopp                    Dexpram-             A small-molecule            Amyotrophic           Phase II data showed the highest dose of
 Biosciences LLC          ipexole              modulator of mito-          lateral               the drug reduced mortality by 68% in the
 (Pittsburgh)                                  chondrial bioener-          sclerosis             second stage of the trial (11/22)
                                               getics
 NeurogesX Inc.           NGX-1998             A topical liquid for-       Postherpetic          Phase II top-line data showed that the trial
 (San Mateo, Calif.)                           mulation of high-           neuralgia             met its primary endpoint of a percentage
                                               concentration                                     change from baseline vs. placebo (11/9)
                                               capsaicin
 Rexahn           Serdaxin                     A neuroprotective           Major depres-         Phase IIb data showed it did not demon-
 Pharmaceuticals                               and antidepressant          sive disorder         strate efficacy compared to placebo over
 Inc. (Rockville,                              candidate                                         an eight-week treatment period (11/7)
 Md.)
 Supernus         SPN-810                      Molindone hydro-            Impulsive             Started a Phase IIb trial (11/29)
 Pharmaceuticals                               chloride                    aggression
 Inc. (Rockville,                                                          associated
 Md.)                                                                      with attention
                                                                           deficit hyper-
                                                                           activity
                                                                           disorder
 INFECTION
 Alnylam          ALN-RSV01                    RNAi therapy                Respiratory           Completed enrollment in a Phase IIb trial
 Pharmaceuticals                                                           syncytial virus       (11/30)
 Inc. (Cambridge,
 Mass.)
 Inviragen Inc.        DENVax                  A dengue vaccine            Dengue fever          Started a Phase II trial (11/29)
 (Fort Collins, Colo.)                         based on an attenu-
                                               ated DEN-2 virus
 Medivir AB               TMC435               An oral HCV prote-          Chronic hepa-         Phase IIb data showed that TMC435 once
 (Huddinge,                                    ase inhibitor               titis C virus         daily in addition to pegylated interferon
 Sweden)                                                                                         and ribavirin produced superior rates of
                                                                                                 sustained virologic response compared to
                                                                                                 standard of care (11/3)

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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 14 OF 32
 Company                  Product              Description                 Indication            Status (Date)#
 (Location)
 SEEK (London)            FLU-v                Vaccine                     Influenza             The vaccine was well tolerated and effec-
                                                                                                 tive against all influenza strains in a Phase
                                                                                                 II challenge trial (11/8)
 Sinovac Biotech          Vaccine              Enterovirus 71 vac-         Hand, foot and        The vaccine had good immunogencity and
 Ltd. (Beijing)                                cine                        mouth disease         a favorable safety profile with no adverse
                                                                                                 events in a Phase II trial (11/10)
 MISCELLANEOUS
 Alexion         Soliris                       Eculizumab                  Atypical hemo- Pivotal Phase II data demonstrated that
 Pharmaceuticals                                                           lytic uremic   ongoing treatment sustained the suppres-
 Inc. (Cheshire,                                                           syndrome       sion of complement-mediated thrombotic
 Conn.)                                                                                   microangiopathy, maintained or further
                                                                                          improved longer-term renal function and
                                                                                          enhanced quality of life (11/11)
 Ampio           Ampion                        A low molecular             Nasal inflam-         A double-blind study in 20 patients
 Pharmaceuticals                               fraction of commer-         mation                showed it was safe and effective for reduc-
 Inc. (Greenwood                               cial albumin                                      ing nasal inflammation (11/11)
 Village, Colo.)
 FibroGen Inc. (San       FG-4592              A hypoxia-inducible Chronic kidney Phase IIb data showed that 96% of patients
 Francisco)                                    factor prolyl hydrox- disease      had an increase in hemoglobin (Hb) of at
                                               ylase inhibitor                    least 1 g/dL, and 93% had an Hb response;
                                                                                  treatment was well tolerated (11/16)
 Kythera                  ATX-101              Formulation of              Submental fat         Data from a long-term follow-up Phase IIa
 Biopharma-                                    deoxycholate, an                                  trial showed more than 90% of initial
 ceuticals Inc.                                endogenous com-                                   responders sustained or improved their
 (Los Angeles)                                 pound that pro-                                   response (11/8)
                                               motes the natural
                                               breakdown of
                                               dietary fat
 ProFibrix BV             Fibrocaps            A dry powder topi-          Liver resection Phase II data showed a 50% reduction in
 (Leiden, the                                  cal fibrin sealant          surgery         mean time to hemostasis, the primary end-
 Netherlands)                                  developed from a                            point, compared to active control (11/16)
                                               mixture of fibrino-
                                               gen and thrombin
 RegeneRx                 RGN-259              Sterile, preservative- Central corne-             Phase II data showed it met statistical sig-
 Biopharma-                                    free eye drop          al fluorescein             nificance after a challenge-controlled
 ceuticals Inc.                                                       staining                   adverse environment in the reduction from
 (Rockville, Md.)                                                                                baseline compared to placebo (11/7)
 Repros                   Androxal             Oral therapy that           Low                   Phase II data showed that 25 mg of
 Therapeutics                                  stimulates the pitu-        testosterone          Androxal showed statistically equivalent
 Inc. (The                                     itary gland                                       efficacy to topical Androgel (11/9)
 Woodlands, Texas)
 Ritter             RP-G28                     A drug designed to          Lactose               Last patient completed treatment in a
 Pharmaceuticals                               stimulate colonic           intolerance           Phase II study (11/18)
 Inc. (Los Angeles)                            growth
 Unigene           Oral PTH                    Oral parathyroid            Osteoporosis          Phase II data showed it achieved the prima-
 Laboratories Inc.                             hormone analogue                                  ry endpoint of increasing bone mineral
 (Boonton, N.J.)                                                                                 density at the lumbar spine (11/10)
 Notes:
 Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.
 # The date indicated refers to the BioWorld Today issue in which the news item can be found.

                To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476.
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MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 15 OF 32

                  Phase III Clinical Trials Update: November 2011
 Company                   Product              Description                  Indication             Status (Date)#
 (Location)
 AUTOIMMUNE
 Genzyme Corp.             Lemtrada             Alemtuzumab                  Relapsing-             Phase III data showed it met both of its
 (Cambridge, Mass.)                                                          remitting              co-primary endpoints (11/15)
                                                                             multiple
                                                                             sclerosis
 NPS                       Natpara              NPSP558; a bioengi-          Hypopara-              Phase III data showed that 53% of
 Pharmaceuticals                                neered replica of            thyroidism             Natpara-treated patients achieved the pri-
 Inc. (Bedminster,                              human parathyroid                                   mary endpoint vs. 2% of placebo-treated
 N.J.)                                          hormone                                             patients (11/8)
 Nuron Biotech             NU100                A recombinant                Relapsing-             Started a pivotal, Phase III trial (11/2)
 Inc. (Exton, Pa.)                              human interferon             remitting multi-
                                                beta-1b                      ple sclerosis
 CANCER
 Amgen Inc.                Xgeva                Denosumab                    Bone metasta-          Phase III data showed that Xgeva signifi-
 (Thousand Oaks,                                                             ses of prostate        cantly prolonged bone metastasis-free
 Calif.)                                                                     cancer                 survival by 4.2 months, delayed bone
                                                                                                    metastasis by 3.7 months and reduced
                                                                                                    symptoms of bone metastasis by 33%
                                                                                                    (11/17)
 Celgene                   Revlimid             Lenalidomide                 Castrate-              Company is terminating the pivotal Phase
 International                                                               resistant              III MAINSAIL trial because the combina-
 Sarl (Boudry,                                                               prostate cancer        tion of docetaxel and prednisone with
 Switzerland)                                                                                       Revlimid did not show a statistically sig-
                                                                                                    nificant improvement in overall survival
                                                                                                    compared to docetaxel and prednisone
                                                                                                    plus placebo (11/28)
 Exelixis Inc.             Cabozantinib         A dual inhibitor of          End-stage cas-         Company was unable to secure an SPA
 (South San                                     MET and VEGFR                trate resistant        and plans to start a Phase III trial by the
 Francisco)                                                                  prostate cancer        end of the year with pain as the primary
                                                                                                    efficacy endpoint; a second Phase III trial
                                                                                                    to evaluate overall survival time will
                                                                                                    launch in 2012 (11/2)
 Medivation Inc.           MDV3100              An oral androgen             Prostate cancer        A independent data monitoring commit-
 (San Francisco)                                receptor antagonist                                 tee found Phase III interim data to be posi-
                                                                                                    tive and recommended the trial be
                                                                                                    stopped so the drug could be offered to
                                                                                                    men receiving placebo (11/4)
 Novocure Inc.             TTF therapy          Tumor treating               Recurrent glio-        Phase III data showed overall survival was
 (Anaheim, Calif.)                              fields therapy               blastoma               9% vs. 7% and 8% vs. 1% for the TTF group
                                                                             multiforme             compared to the standard of care at two
                                                                                                    and three years of follow-up, respectively
                                                                                                    (11/22)
 Talon               Marqibo                    Vincristine sulfate          Newly                  Enrolled the first patient in the Phase III
 Therapeutics Inc.                              liposomes injection          diagnosed              study of Marqibo (11/30)
 (San Mateo, Calif.)                                                         aggressive
                                                                             non-Hodgkin’s
                                                                             lymphoma



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                                              Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
MONDAY, DEC. 12, 20 1 1                                        BIOWORLD® INSIGHT                                                            PAGE 16 OF 32
 Company                  Product              Description                  Indication              Status (Date)#
 (Location)
 CARDIOVASCULAR
 Almirall SA              Aclidinium           Fixed-dose combi-            Chronic                 Began a Phase III trial (11/17)
 (Barcelona, Spain)       bromide and          nation via the               obstructive pul-
 and Forest               formoterol           Genuari inhaler              monary disease
 Laboratories Inc.        fumarate
 (New York)
 Gilead Sciences          Ranexa               Ranolazine                   Chronic angina          Started a Phase III trial (11/8)
 Inc. (Foster City,
 Calif.)
 Inspiration              OBI-1                An intravenous         Congenital                    Started patient enrollment in the seconf
 Biopharma-                                    recombinant por-       hemophilia A                  of two pivotal studies (11/28)
 ceuticals Inc.                                cine Factor VIII prod-
 (Laguna Niguel,                               uct
 Calif.)
 Omthera                  Epanova              Triglyceride-                High                    Completed enrollment in a pivotal, Phase
 Pharmaceuticals                               lowering therapy             triglycerides           III trial (11/30)
 Inc. (Bedminster,
 N.J.)
 Santarus Inc. (San Rhucin                     Recombinant                  Hereditary              A retrospective analysis showed that
 Diego)                                        human C1 inhibitor           angioedema              median time to primary endpoint of onset
                                                                                                    of symptom relief was 60 minutes and
                                                                                                    median time to minimal symptoms was
                                                                                                    240 minutes (11/7)
 CENTRAL NERVOUS SYSTEM
 Alexza                   Adasuve              Staccato loxapine            Schizophrenia           Phase III data showed a 40% or greater
 Pharmaceuticals                                                            or bipolar I            decrease from baseline in the total PANSS-
 Inc. (Mountain                                                             disorder                Excited Component score; another analy-
 View, Calif.)                                                                                      sis showed a statistically significant
                                                                                                    improvement over placebo using the
                                                                                                    Clinical Global Impression-Improvement
                                                                                                    scale (11/9)
 Alkermes plc             Vivitrol             Naltrexone                   Opioid                  Data showed sustained efficacy in those
 (Dublin)                                                                   dependence              receiving Vivitrol in combination with
                                                                                                    psychosocial treatment for 18 months
                                                                                                    (11/10)
 Nabi Biopharma-          NicVAX               Smoking cessation            Smoking                 A second Phase III trial failed to meet its
 ceuticals Inc.                                vaccine                      cessation               primary endpoint of abstinence from cig-
 (Rockville, Md.)                                                                                   arettes; the vaccine was well tolerated
                                                                                                    (11/8)
 Targacept Inc.           TC-5214              Nicotinic channel            Major depres-           Failed to meet its primary endpoint of
 (Winston Salem,                               modulator to nor-            sive disorder           change on the Montgomery-Asberg
 N.C.)                                         malize cholinergic                                   Depression Rating Scale following eight
                                               tone                                                 weeks of treatment in a Phase III trial (11/9)
 Vivus Inc.               Qnexa                Phentermine/topira- Obesity                          Results from the 56-week EQUIP study in
 (Mountain View,                               mate                                                 1 ,267 patients showed an average weight
 Calif.)                                                                                            loss of 14.4% of initial body weight, plus
                                                                                                    improvements in blood pressure, glucose,
                                                                                                    triglycerides and cholesterol (11/7)



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CNDO 11-12-11 BioWorld Insight

  • 1. ® MONDAY VOL.19, NO. 50 Your Weekly Source for Market and Financial Views DEC. 12, 2011 PAGE 1 OF 32 In Geron’s Wake Money Raised By Biotech Deals, Data Spark Stem Cell In 2011 Vs. 2010 Interest, But Hurdles Remain Jan. 1 – Dec. 9, 2011: $22,777.24M By Trista Morrison Jan. 1 – Dec. 9, 2010: $18,987.47M Editor SAN DIEGO – Geron Inc.’s exit from the embryonic stem 11,000 cell field last month shocked many in the biotech industry 10,000 9,792 – but not the regenerative medicine experts attending the 8,634 9,000 recent 2011 Stem Cell Meeting on the Mesa. 7,863 Some attendees muttered that Geron had never been 8,000 able to effectively balance the conflicting risk-reward 7,000 6,700 profiles of its stem cell business and its cancer business. Millions 6,000 Others whispered that the writing had been on the wall since the departure of longtime CEO Thomas Okarma in 5,000 4,351 4,425 February. Still others wondered if perhaps the preliminary 4,000 data from Geron’s Phase I spinal cord injury trial – the world’s first of a human embryonic stem cell therapy – 3,000 weren’t as good as had been hoped. 2,000 Geron has maintained the latter isn’t the case – the 1,000 company blamed its withdrawal from the stem cell space on capital scarcity and uncertain economic conditions. (See 0 BioWorld Today, Nov. 16, 2011 .) Public Public/ Private Of f erings Other* Biotechs Regardless of the reason, regenerative medicine * Includes financings of public biotech firms with the exceptions of insiders don’t view Geron’s move as an indication that public offerings and certain investments from corporate partners. the stem cell industry is on shaky ground – at least, not any shakier than it’s ever been. If anything, data and deal- “has been sorted out,” he said, and advances in research flow indicate increasing interest in stem cells, although tools have improved the consistency of embryonic stem maintaining that momentum will require creativity on cell work as well. On the embryonic side, Geron also blazed several fronts. a regulatory trail with the FDA by getting into the clinic. “The greatest obstacle now is to deliver compelling Kicking into Gear clinical proof of concept data,” said Gil Van Bokkelen, CEO of Mahendra Rao, director of the National Institutes of Athersys Inc. and chairman of the Alliance for Regenerative Health’s Center for Regenerative Medicine, noted that the Medicine. “That’s what investors are looking for; that’s stem cell field has been significantly derisked over the past few years. Most of the technical risk with adult stem cells See Stem Cells, page 4 BIOTECH SHORT SELLING UP SLIGHTLY, BUT NOT ALL BAD ...................... 2 THIS WEEK IN FORM 10 AS IPO ALTERNATIVE? CORONADO WOULD DO IT AGAIN ........ 3 MONEY RAISED BY BIOTECH IN 2011 ................................................... 4 BIOWORLD WEEK IN REVIEW ................................................................................ 5 WORD ON THE STREET, WEEK IN WASHINGTON ..................................... 6 INSIGHT PHASE I, II & III CLINICAL TRIALS UPDATE: NOVEMBER 2011 .... 7, 11, 15 CONFERENCE DATA ...................................................................... 19-25 NON-U.S. CLINICAL TRIALS DATA; FDA ACTIONS ......................... 26, 28 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 2. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 2 OF 32 A Difference of Opinion Despite continued volatility in the market, the Biotech Short Selling Up measurements of short activity have returned to normal levels. Slightly, But Not All Bad There has, however, been a slight widening of the short By Brian Orelli interest as a percent of equity over the last two and a half BioWorld Insight Contributing Writer years between biopharma and Nasdaq stocks, with the Short sellers are generally considered blasphemers by biopharma stocks closer to 1 .5 percentage points higher biotech firms. And for good reason; short sellers borrow than the Nasdaq stocks, up from the previously mentioned shares in order to sell them, which puts negative pressure on mean of about 1 percentage point. Grimaldi offered up a the stock. The only way shorts make money is if shares go couple of potential explanations: “It may mean that they’re down. betting against the sector or there may just be a larger But biotechs don’t have much to complain about, according number of companies where it would be profitable to put to data compiled by BMO Capital Markets. Short sellers are on a short position.” only slightly more active in the biopharma sector than they Not every short position is made by an investor who are for the entire Nasdaq. Looking at the short interest as a believes the shares are overvalued. Short sales are used percent of equity float since the beginning of 2007, the 302 to hedge long positions in either the individual stock, the publicly traded biopharmas used in the study had a mean of sector, or the entire investor’s portfolio. They can also be about 6.6 percent of their available shares sold short, which is used as a tax deferral strategy. only about a percentage point higher than the short interest Shorts also increase liquidity of shares, which can be for all the Nasdaq stocks. helpful for biotechs. Higher liquidity, for instance, helps Considering the large number of binary events in biotech – data read-outs, FDA decisions, etc. – Annette Grimaldi, See Shorts, page 6 managing director at BMO, thought the absolute difference might have been larger. One possible reason is that the binary events also detract short investors because the losses on a short position are theoretically limitless, unlike a long position where the most an investor can lose is the initial investment. BioWorld is now The means of the short interest ratio – dividing the short interest position by the average daily trading volume – were on Twitter! nearly identical at 7.8 and 7.6 for Nasdaq and biopharma stocks, respectively. In other words, it would take almost eight days for the shorts to cover all their outstanding short sales at Stay Connected, the average trading volume. Follow Us on Twitter! Both the short interest as a percent of equity float and the short interest ratio spiked in the middle of 2008 during the peak of the recession. That’s not surprising as both www.twitter.com/bioworld measurements describe how safe it is to put on a short position. As the numbers increase, it becomes harder to unwind the short. BIOWORLD® INSIGHT (ISSN 1541-0579) is published every Monday by AHC Media, 3525 Piedmont Road, Building Six, Suite 400, Atlanta, GA 30305. Opinions expressed are not necessarily those of this publication. Mention of products or services does not con- stitute endorsement. BIOWORLD® and BIOWORLD® INSIGHT are trademarks of AHC Media, a division of Thompson SUBSCRIBER INFORMATION Media Group LLC. Copyright © 2011 AHC Media. All Rights Reserved. No part of this publication may be repro- Please call (800) 888-3912 to duced without the written consent of AHC Media. (GST Registration Number R128870672) subscribe or if you have fax trans- ATLANTA NEWSROOM: Executive Editor: Lynn Yoffee. mission problems. Outside U.S. and Managing Editor: Jennifer Boggs. Canada, call (404) 262-5476. Our customer service hours are 8:30 Editor: Trista Morrison. a.m. to 6:00 p.m. EST. Database Editor: Karen Pihl-Carey. BUSINESS OFFICE: Senior Vice President/Group Publisher: Donald R. Johnston. EDITORIAL Lynn Yoffee: (404) 262-5408 Senior Editor: Michael Harris. Trista Morrison: (858) 901-4785 Managing Editor: Amanda Lanier. Donald R. Johnston: (404) 262-5439 Product Marketing Manager: Sarah Cross. Internet: http://www.bioworld.com Account Representatives: Matt Hartzog, Chris Wiley. DISPLAY ADVERTISING: For ad rates and information, please call Stephen Vance at (404) 262-5511. or e-mail him at stephen.vance@ahcmedia.com. REPRINTS: For photocopy rights or reprints, call our reprints department at (404) 262-5547. PRESS MATERIALS: Send all press releases and related information to newsdesk@bioworld.com. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 3. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 3 OF 32 Weighing Pros, Cons Unlike in an IPO, Coronado didn’t have investment Form 10 as IPO Alternative? bankers trotting management around to meet with institutional investors and raise awareness. “With Form 10, Coronado Would Do It Again you essentially have to do it yourself,” Sandage explained, By Trista Morrison adding that he and chief operating officer Noah Beerman Editor have been “on the road nonstop” talking to investors and Four months ago, Coronado Biosciences Inc. embarked on analysts, trying to get the word out. a route to going public not often seen in the biotech industry: For now, trading volume among Coronado’s largely the Form 10 pathway. (See BioWorld Today, July 20, 2011 .) retail base is low, but Sandage isn’t overly concerned, noting In essence, the Form 10 pathway offers an alternative to the that “IPOs don’t trade that much after the first week or two initial public offering (IPO) or reverse merger, seeking to avoid either.” And although Coronado’s price has fallen, Sandage the market volatility and high transaction costs of the former is encouraged by the fact that the firm was recently picked as well as the trading restrictions and investor baggage of the up by Roth Capital Partners with a buy rating and a $27 price latter. A private firm looking to use the pathway to go public target. He’s also optimistic that more analyst coverage and simply files a Form 10 with the SEC to become a public reporting interest from institutional investors will come once the firm company, files an S-1 to register its shares, and applies to trade transfers to Nasdaq, a process that is already underway. those shares on a market. (See BioWorld Insight, July 25, 2011 .) Beerman also believes Coronado’s stock price Coronado got started down the Form 10 road because will benefit from the achievement of some near-term such filings are required for private companies with 500 or milestones. The company recently filed an investigational more stockholders of record. The rule most often affects large new drug application for lead program CNDO-201 , pig private tech firms like Facebook, but Coronado’s angel-backed whipworm eggs, and a Phase I trial is underway. Safety business model made it one of the rare few private biotechs data are expected in the first quarter of 2012, and a Phase II to hit the stockholder threshold. Although a bill is pending in trial for Chron’s disease is expected to start in the second the House that may raise that threshold, under current law, quarter. Meanwhile Coronado’s second program, which Coronado had to become a reporting company. involves ex-vivo activation of NK cells, is poised to start a Coronado did not, however, have to start publicly Phase I/II trial in acute myeloid leukemia early next year. trading its shares – but the company was interested in It remains to be seen whether or not other biotechs accessing the public markets. President and CEO Bobby follow in Coronado’s footsteps and use the Form 10 Sandage said Coronado had considered a reverse merger pathway to go public. They may soon have yet another and even went as far as to identify a suitable shell company IPO alternative: a bill that has passed the House and is to merge into, but new restrictions from the SEC made that now in the Senate would raise the Regulation A exemption option less appealing. Meanwhile, although five biotechs to $50 million, providing privately held biotechs with the have jumped into the IPO queue in the last few months, the opportunity to raise money and start trading their shares window remains far from what most would consider open. without filing a Form 10 to become a public reporting (See BioWorld Today, Nov. 11 , 2011 .) company. (See BioWorld Insight, Nov. 21 , 2011 .)  Hence Coronado opted for the Form 10 route. Sandage said the process is not well understood and “even with experienced SEC lawyers we were sort of figuring things out as we went along,” but he believes it was the right decision and would do it again with future start-ups. Coronado debuted on the OTCQB last month at $11 per ADVERTISE share. That price was market-driven, determined not by a team of investment bankers negotiating with institutional investors, but simply by what a buyer was willing to pay and HERE a seller was willing to accept. Interestingly, Coronado’s price was higher than that of three-quarters of the biotechs to price ...and reach high-level IPOs this year, even though the Burlington, Mass.-based firm is biotechnology professionals every week! barely in the clinic. Since its pricing, however, Coronado’s shares For advertising opportunities in (OTCQB:CNDO) have dropped 34 percent to $7.25, as of mid BioWorld Insight, please contact last week. That’s not necessarily unusual for a newly public biotech – several members of this year’s IPO class have Stephen Vance at (404) 262-5511 fallen just as hard, although as a group they are down about or stephen.vance@ahcmedia.com 8 percent. The drop does, however, illustrate some of the challenges of taking the Form 10 route. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 4. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 4 OF 32 Another sign of momentum can be seen in private Money Raised By Biotech: financing for stem cell and regenerative medicine firms. Jan. 1 - Dec. 9, 2011 Four such firms have completed private offerings this year, according to data from Roth Capital Partners, and three of them brought in at least $30 million. Additionally, 19% 43% three firms in the field – Argos Therapeutics Inc., TVAX ($4,351M) ($9,792M) Biomedical Inc. and Verastem Inc. – have filed to go public, and Advanced BioHealing was in the initial public offering queue as well before its acquisition. Not Out of the Woods So is the financial situation for regenerative medicine companies really that much worse than for biotech as a whole, as Geron’s retraction indicated? It’s somewhat hard to believe, sitting in the brand-spanking-new $127 million laboratories of the Sanford Consortium for Regenerative Medicine, listening to so many examples of progress and success. But according to Geoff Whitehead, principal with Flagship Ventures, the answer is yes. The outlook for stem cell firms is not quite as dire on 38% Public Offerings 43% ($9,792M) the private equity side. Venture capitalists are looking ($8,634M) for transformative ideas, large markets and compelling Public/Other 38% ($8,634M) entrepreneurs, so the fact that a lot of stem cell work is early Private Biotechs 19% ($4,351M) stage and hard to value “doesn’t really factor into things,” Whitehead said. Flagship has three regenerative medicine firms in its portfolio. But on the other hand, Whitehead noted that “early Stem Cells stage VCs like to invest in pioneers,” and the stem cell field’s Continued from page 1 pioneers don’t have the best track record thus far. He then what pharma is looking for. Once that data gets delivered, it ticked off a dozen reasons why it would be easier for the will change the mindset in a big way.” average VC to say “no” to a stem cell investment than “yes.” In the adult stem cell space, data is starting to trickle Stem cell start-ups do have the benefit of alternative out. Morrie Ruffin, managing director of the Alliance for options like grant funding – the California Institute of Regenerative Medicine, noted that while the media were busy Regenerative Medicine (CIRM) has been doling out money reporting on Geron, many missed the fact that Mesoblast for several years now. But Edward Lanphier, president Ltd.’s allogeneic adult stem cell product Revascor significantly and CEO of Sangamo BioSciences Inc., noted that the only reduced cardiovascular deaths in a randomized, placebo- companies able to go public these days are in Phase III, and controlled Phase II trial, and Aastrom Biosciences Inc.’s “is CIRM going to take someone to Phase III? I don’t think so. autologous expanded cell therapy ixmyelocel-T significantly There’s a need for venture capital. I’m not optimistic that it’s reduced treatment failures in a randomized, placebo- going to be met, but there’s a need.” controlled Phase II critical limb ischemia trial. (See BioWorld Even if a stem cell company does get public, that Today, Nov. 16, 2011 .) doesn’t mean their fortunes will improve. Lisa Walters- There’s been good news on the partnering front as well. Hoffert, managing director with Roth, presented an analysis Every panel at the conference paid homage to Mesoblast’s showing that just nine public stem cell and regenerative $350 million stem cell deal with Cephalon Inc. (now part of medicine firms have raised money this year – less than $140 Teva Pharmaceutical Industries Ltd.), which carried a whopping million altogether, and with a hefty average 67.5 percent $130 million up front, as well as to Shire plc’s $750 million buy- warrant coverage. Since their offerings, the firms have seen out of regenerative medicine firm Advanced BioHealing Inc. their shares fall an average of 24 percent, and only one (See BioWorld Today, May 19, 2011, and Dec. 9, 2010.) company – StemCells Inc. – is in the green. “Things move forward at a certain rate and then kick into Further, despite the oft-mentioned Mesoblast and gear,” said Robert Preti, president and chief scientific officer Advanced BioHealing deals, business development in the of Progenitor Cell Therapy LLC, which itself was recently the space has yet to catch fire. subject of some business development activity, having been “We spend a lot of time talking to pharma,” said acquired by NeoStem Inc. NeoStem also acquired stem cell firm Sangamo’s Lanphier. “As it relates to cell therapy, there is Amorcyte Inc. earlier this year. (See BioWorld Today, July 18, 2011 .) See Stem Cells, page 5 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 5. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 5 OF 32 WEEK IN REVIEW Financings Dendreon Corp. raised $125 million by monetizing its share of royalties on Victrelis (boceprevir). EMBL Ventures GmbH closed a $53.5 million fund for German life science companies. Forbion Capital Partners closed its second FCF 1 Co-Investment Fund at about $50 million. Gilead Sciences Inc. priced $3.7 billion worth of senior unsecured notes in a public offering. Deals Biogen Idec Inc. entered a $300 million joint venture with Samsung for biosimilars. Medicis Pharmaceutical Corp. acquired the assets of Graceway Pharmaceuticals LLC for $455 million. Molecular Partners AG signed an immunology discovery deal with Janssen Biotech Inc. Momenta Pharmaceuticals Inc. acquired Virdante Pharmaceuticals Inc.’s protein sialylation programs. . . . And More See BioWorld Today for coverage of the San Antonio Breast Cancer Symposium. Affymax Inc.’s peginesatide got a thumbs up from an FDA advisory committee. Curemark LLC reported positive Phase III data with CM-AT in children with autism. Genentech Inc./Roche AG’s pertuzumab had good Phase III breast cancer data. Theratechnologies Inc. is halting its chronic obstructive pulmonary disease muscle wasting program and downsizing by 60 percent. Unigene Laboratories Inc.’s parathyroid hormone was dropped by partner GlaxoSmithKline plc. Stem Cells “high-risk work” that neither grant-funded researchers nor Continued from page 4 companies can stomach. Nonprofits should also focus on the translational work that academics aren’t interested in interest, but there is a higher bar. Whether it’s safety, efficacy, because it won’t result in a published paper – the “work that cost of goods analysis . . . you’ve got to paddle your own canoe is repetitive but essential to derisk the program to a point a lot longer.” where investors or pharma can pick it up,” she said. Matthias Steger, global head of research and technology Meanwhile, history (and Geron) has proven the partnering with Roche AG, provided further perspective. traditional drug development business model isn’t a While he refuted the notion that big pharma is not involved particularly good fit for stem cell firms. Whether public or in stem cells, pointing to Roche’s own significant investment, private, the firms that have managed to stay in business he cautioned that big pharma “can just not afford running are those that have, more often than not, found alternative into big failures. We have to sort out the science. It would be a sources of funding. mistake to rush. I think we are making progress, but we have Sangamo, for example, has outlicensed its zinc finger to be a little bit patient.” protein technology in non-core fields like agriculture and research tools. Cytori Therapeutics Inc. is bringing in Just Keep Paddling revenue by selling its Celution adult stem cell processing The trick will be for stem cell firms to figure out how they system in Europe, where it has received a CE Mark, while it can afford to patiently wait for the catalyst that captures big navigates the U.S. regulatory path. Organovo Inc. uses its pharma’s attention, or the proof-of-concept data that drive platform to create disease models for partners, as well as public market interest. in its own drug development. Neostem generates revenues The experts at the conference said it will require an from a cell therapy manufacturing business, a stem cell unprecedented level of public-private partnership. “It can’t bank and several initiatives in China, which help support be industry alone, or academics alone, or government alone the cost of developing its cell therapy pipeline. – there must be collaboration,” said NIH’s Rao. He added The approaches are unconventional, but Greg Lucier, that there are far more stem cell trials being conducted by chairman and CEO of Life Technologies Corp., thinks academics than by industry, but the former have “no idea how that’s what the regenerative medicine industry needs. “I to take it to the next level.” That’s where government needs to do believe this is an area where we should be pushing the focus its efforts, he said. frontiers,” he said. “We’re positive, we’re confident and we Susan Solomon, CEO of the New York Stem Cell are investing in this area. The horizon we see is near term Foundation, added that nonprofits can play a role by doing the and incredibly bright.”  To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 6. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 6 OF 32 WORD ON THE STREET “If you’re a great company with great promise, there’s money out there.” – Geoff Whitehead, principal with Flagship Ventures, on funding difficulties for stem cell firms “The world of pharma is waking up [to the fact] that this is going to be the next frontier.” – Greg Lucier, chairman and CEO of Life Technologies Corp., on stem cells “You want to have Track 1 , 2, 3, 4 and 5 – if you only have Track 1 and Track 5, no one wants to give it a shot.” – William Hicks, partner with Mintz Levin, on the need to have multiple avenues to going public “The hurdle rates for companies in development are being set higher than they have been in the past. Having said that, we love being in this industry.” – Anthony Marucci, president and CEO, Celldex Therapeutics Inc. WEEK IN WASHINGTON The FDA’s latest proposed user fee structure for biosimilars will charge 10 percent of the new drug application fee, plus a development fee. The U.S. Patent and Trademark Office and China’s State Intellectual Property Office will expedite their patent examinations, which should allow applicants to obtain corresponding patents faster in each country. Shorts While shorts might be best ignored from an industry Continued from page 2 perspective, Grimaldi thinks individual firms should firms sell more shares in at-the-market (ATM) offerings, a probably consider why investors are shorting the shares. public offering alternative that has slowly gained traction over “Especially large short interest is a signal that differs from the past few years. Inhibitex Inc. raised $20 million through what management is saying. For that particular company, an ATM last month. (See BioWorld Insight, Sept. 7, 2009.) they should look to see if there are any underlying messages And short sellers can actually have a positive effect on in that position.” shares if a company produces positive news. A short squeeze China Biologic Products Inc., Cumberland Pharma- occurs as the short sellers cover their sales by buying back ceuticals Inc., MannKind Corp., BioTime Inc., and Oncolytics shares, amplifying the upward movement of the stock in Biotechnology Inc. currently top the list of companies with response to the good news. the highest short interest.  READING A HAND-ME-DOWN? If you’re reading this issue, but not a subscriber, please call us to start receiving your very own copy. Or, for even more convenience, subscribe to our online version or have it e-mailed directly to you! Authorized copying privileges and multi-user access for your entire office are available at discounted rates. Simply call 1-800-688-2421 or 1-404-262-5476 for more information! To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 7. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 7 OF 32 FDA Approvals In November Company Drug Indication Bristol-Myers Squibb Co. Erbitux Head and neck cancer EUSA Pharma Inc. Erwinase Acute lymphoblastic leukemia Incyte Corp. Jakafi Myelofibrosis IntelGenx Corp. CPI-300 Major depressive disorder Millennium Velcade Updated label for multiple myeloma Pacira Pharmaceuticals Inc. Exparel Postsurgical pain relief Regeneron Pharmaceuticals Inc. Eylea Wet age-related macular degeneration Spectrum Pharmaceuticals Inc. Zevalin Removed bioscan requirment for non-Hodgkin’s lymphoma Transcept Pharmaceutical Inc. Intermezzo Insomnia Phase I Clinical Trials Update: November 2011 Company Product Description Indication Status (Date)# (Location) CANCER Bavarian Nordic CV-301 Off-the-shelf immu- Metastatic breast A trial with 26 heavily pretreated patients A/S (Kvistgard, notherapy or ovarian cancer who received monthly vaccinations Denmark) showed a median time to progression of 2.5 months in the breast cancer group, and median overall survival was 13.7 months (11/10) Celldex CDX-1127 A co-stimulatory Malignant solid Started a Phase I study (11/9) Therapeutics Inc. molecule on T cells tumors or hema- (Needham, Mass.) tologic cancers CureVac GmbH CV9201 mRNA-based can- Non-small-cell Phase I/IIa data showed the drug was well (Tuebingen, cer vaccine lung cancer tolerated and biologically active (11/8) Germany) CytRx Corp. (Los INNO-206 A tumor-targeting Sarcoma Phase Ib/II trial data determined a maxi- Angeles) conjugate of doxo- mum tolerated dose of the drug deliver- rubicin ing doxorubicin at the equivalent of three and a half times standard doxorubicin doses (11/1) Hutchison Epitnib An orally active Cancer Started the first-in-human Phase I trial MediPharma Ltd. (HMPL-813) small molecule (11/7) (London) inhibitor targeting the epidermal growth factor receptor Infinity IPI-145 Oral inhibitor of Advanced Started two Phase I trials (11/1) Pharmaceuticals phosphinositide-3- hematologic Inc. (Cambridge, kinase delta and malignancies Mass.) gamma Lentigen Corp. LG631 Gene therapy Cancer Started a Phase I trial (11/15) (Gaithersburg, Md.) Senesco SNA01-T Intravenous infusion Multiple Initiated patient dosing in its Phase Ib/IIa Technologies Inc. myeloma study (11/4) (Bridgewater, N.J.) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 8. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 8 OF 32 Company Product Description Indication Status (Date)# (Location) Silence Atu027 siRNA drug Solid tumors Phase I data showed 37% of patients had Therapeutics plc stable disease after treatment (11/9) (London) CARDIOVASCULAR Cardiome Vernakalant Oral therapy Atrial fibrillation Recently completed an additional multi- Pharma Corp. ple rising-dose Phase I study and data (Vancouver, British showed the drug was well tolerated (11/7) Columbia) and Merck & Co. Inc. (Whitehouse Station, N.J.) Nile Cenderitide Subcutaneous Chronic heart Phase I data showed that weight-based Therapeutics Inc. infusion failure doses achieved target PK levels when (San Mateo, Calif.) delivered by subcutaneous pump, and that it was well tolerated, bioavailable and reduced PK variability compared to a fixed-dose regimen (11/17) Pozen Inc. (Chapel PA32540 A combination of Gastrointestinal Phase I data showed it was associated Hill, N.C.) 325 mg aspirin and risk in patients with greater platelet inhibition when 40 mg immediate- who require dual dosed 10 hours apart from Plavix com- release omeprazole antiplatelet ther- pared to synchronous administration of apy and gastro- aspirin, clopidogrel and delayed-release protection omeprazole at day seven (11/15) CENTRAL NERVOUS SYSTEM Acadia AM-831 A small molecule Schizophrenia FDA cleared it to begin a Phase I trial (11/8) Pharmaceuticals that combines mus- Inc. (San Diego) carinic m1 partial agonism with dopa- mine D2 and sero- tonin 5-HT2A antagonism Anavex Life Anavex 2-73 The first of a new Alzheimer’s Completed a Phase I single ascending Sciences Corp. class of oral drugs disease dose trial of Anavex, showing it was well (Hoboken, N.J.) tolerated below the 55 mg to 60 mg dose with only mild adverse events (11/15) Cara CR845 A peptide-based Acute postopera- Started its first Phase I trial of an oral for- Therapeutics Inc. kappa opioid ago- tive pain mulation (11/23) (Shelton, Conn.) nist Neuralstem Inc. Stem cell Stem cell therapy Amyotrophic Dosed its first patient in the trial (11/28) (Rockville, Md.) therapy used in the cervical lateral sclerosis region of the spine NeuroDerm Ltd. ND0611 Administered sub- Parkinson’s Met all primary and secondary endpoints (Ness Ziona, Israel) cutaneously by a disease in a Phase I/II trial (11/10) dermal patch Probiodrug AG PQ912 A glutaminyl Alzheimer’s Phase I data demonstrated it was safe and (Halle, Germany) cyclase inhibitor disease well tolerated (11/15) Selecta SEL-068 A nicotine vaccine Smoking cessa- Started a Phase I trial (11/22) Biosciences Inc. tion and relapse (Watertown, Mass.) prevention To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 9. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 9 OF 32 Company Product Description Indication Status (Date)# (Location) DIABETES Akebia AKB-9778 A human protein Diabetic macular Started a Phase I trial (11/22) Therapeutics Inc. tyrosine phospha- edema and dia- (Cincinnati) tase beta inhibitor betic retinopathy Concert CTP-499 Controlled-release Diabetic Phase I data showed it was well tolerated Pharmaceuticals version; analogue nephropathy at single doses up to and including 1 ,800 Inc. (Lexington, of 1-([S]-5- mg (11/15) Mass.) hydroxyhexyl)-3,7- dimethylxanthine Convergence CNV2197944 A calcium channel Chronic pain Initiated a Phase I trial (11/22) Pharmaceuticals blocker Ltd. (Cambridge, UK) DARA DB959 A peroxisome pro- Type II diabetes Phase Ib data showed a safety profile BioSciences Inc. liferator activated comparable to placebo when given as (Raleigh, N.C.) receptor-delta/ once-daily doses for seven days and was gamma agonist safe and well tolerated throughout the 40-fold dose range tested (11/2) Isis ISIS-GCGRRx Antisense drugs Type II diabetes Started Phase I studies (11/3) Pharmaceuticals and ISIS- Inc. (Carlsbad, GCCRRx Calif.) PhaseBio Glymera A recombinant glu- Hyperglycemia Phase I/IIa data demonstrated statistically Pharmaceuticals cagon-like peptide-1 in Type II significant reductions in fasting glucose Inc. (Malvern, Pa.) analogue diabetes glycemic load following meal tolerance testing and in average daily glucose (11/16) INFECTION AVIR Green Hills deltaFLU Intranasal flu Influenza The vaccine was well tolerated and signif- Biotechnology vaccine icantly increased influenza-specific anti- AG (Vienna, bodies in a Phase I/II trial (11/8) Austria) Inhibitex Inc. INHX-189 Monotherapy; Hepatitis C virus Data showed INX-189 dosed at 100 mg (Atlanta) nucleotide poly- once daily for seven days in combination merase inhibitor with ribavirin resulted in a median HCV RNA reduction from baseline of -0.379 log10 (11/30) Inovio SynCon Avian influenza Avian flu A single intradermal electroporation Pharmaceuticals vaccine boost of its vaccine generated hemagglu- Inc. (Blue Bell, Pa.) tination inhibition titers against six differ- ent unmatched strains of H5N1 and it generated a fourfold or greater rise in HAI titers in 50% of boosted subjects in a Phase I study (11/18) Novacta NVB302 An oral treatment Hospital- Started dosing the first healthy volun- Biosystems Ltd. acquired teers in a Phase I trial (11/3) (London) Clostridium difficile infections To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 10. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 10 OF 32 Company Product Description Indication Status (Date)# (Location) MISCELLANEOUS Aeolus AEOL 10150 A broad-spectrum Acute radiation Pilot study results of AEOL 10150 and Pharmaceuticals catalytic antioxi- syndrome G-CSF drug Neupogen confirmed that it Inc. (Mission Viejo, dant designed to does not interefer with the positive Calif.) neutralize reactive effects of Neupogen on the hematopoiet- oxygen and nitro- ic syndrome (11/2) gen species Akebia AKB-6548 Oral hypoxia- Anemia associat- Phase Ib/IIa data showed it was well toler- Therapeutics Inc. inducible factor- ed with chronic ated and enhanced erythropoiesis (11/16) (Cincinnati) prolyl hydroxylase kidney disease inhibitor AlloCure Inc. AC607 A bone marrow- Acute kidney Phase I data showed it was safe and well (Burlington, Mass.) derived mesenchy- injury tolerated, and treated subjects experi- mal stem cell enced a lower incidence of AKI, reduced therapy length of hospital stay and reduced hos- pital readmission rates compared to a cohort of historical controls (11/14) Alnylam ALN-TTR01 An RNAi therapeu- TTR-mediated Phase I data showed statistically signifi- Pharmaceuticals tic targeting trans- amyloidosis cant reductions in serum TTR protein lev- Inc. (Cambridge, thyretin els in ATTR patients (11/22) Mass.) Bellicum CaspCIDe Technology used to Graft-vs.-host Results of the first clinical study showed Pharmaceuticals eliminate donor T disease it worked quickly and effectively without Inc. (Houston) cells compromising the function of the cell (11/4) Chimerix Inc. CMX001 A lipid-antiviral- Kidney disease Phase I/II data showed it was generally (Durham, N.C.) conjugate designed safe and well tolerated when adminis- to deliver cidofovir- tered to renal transplant and hematopoi- diphosphate etic stem cell transplant patients (11/14) Geron Inc. (Menlo hESC therapy Human embryonic Acute spinal Company is stopping recruitment to its Park, Calif.) stem cell therapy injury Phase I trial due to economic conditions (11/16) Pluristem PLX-PAD Cells derived from Critical limb Phase I data showed that it met all the Therapeutics Inc. the firm’s PLX ischemia endpoints, demonstrating a safe immuno- (Haifa, Israel) (PLacental eXpand- logic profile at all dosage levels and ed) platform showing potential efficacy (11/4) Provesica Ltd. XEN-D0501 An antagonist of Overactive Phase I data showed it was safe and well (Cambridge, UK) TRPV1 bladder tolerated (11/28) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 11. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 11 OF 32 Phase II Clinical Trials Update: November 2011 Company Product Description Indication Status (Date)# (Location) AUTOIMMUNE Adeona Trimesta Oral estriol candi- Multiple It will be tested in a Phase II trial evaluating Pharmaceuticals date sclerosis its potential effect on cognitive dysfunc- Inc. (Ann Arbor, tion in female MS patients (11/14) Mich.) Avanir AVP-923 An NMDA antago- Neuropathic Enrolled the first patient in a Phase II trial Pharmaceuticals nist and sigma-1 pain in multi- expected to enroll 400 patients worldwide Inc. (Aliso Viejo, agonist ple sclerosis (11/4) Calif.) Galapagos NV GLPG0634 JAK1 inhibitor Rheumatoid Achieved the primary endpoint of signifi- (Mechelen, arthritis cant improvement in the signs and symp- Belgium) toms of RA in a Phase IIa trial (11/28) KAI KAI-4169 Long-acting intrave- Secondary Phase II data showed it was effective, with Pharmaceuticals nous calcium sens- hyperparathy- 33% and 49% reductions in parathyroid Inc. (South San ing receptor agonist roidism hormone (PTH) from baseline in the 5-mg Francisco) and 10-mg dose groups, respectively (11/14) Opexa Tovaxin A T-cell therapy Relapsing- Phase IIb data showed it was well tolerated, Therapeutics remitting mul- with no serious adverse events, and dem- Inc. (The tiple sclerosis onstrated encouraging results in the reduc- Woodlands, Texas) tion of the annualized relapse rate and improvement in disease progression (11/15) SuppreMol SM101 A soluble version of Lupus Started dosing in a Phase IIa trial (11/29) GmbH the Fc gamma recep- (Martinsried, tor IIb Germany) CANCER Biothera Inc. Imprime PGG An immunomodula- Non-small-cell Completed enrollment of 90 patients in its (Eagan, Minn.) tor drug lung cancer Phase II trial in combination with Erbitux (11/18) Celldex CDX-110 Rindopepimut EGFRvIII- Phase II data showed final overall survival Therapeutics positive glio- of 24.6 months from diagnosis, compared Inc. (Needham, blastoma to 15.2 months for a historic control (11/22) Mass.) Exelixis Inc. Cabozantinib MET and VEGFR2 Hormone Initiated a Phase II trial (11/22) (South San inhibitor receptor-posi- Francisco) tive breast can- cer with bone metastases Galena NeuVax Vaccine Breast cancer Phase II data showed that it may provide a Biopharma Inc. meaningful clinical benefit in patients with (Lake Oswego, less aggressive forms of the disease (11/7) Ore.) Jennerex Inc. JX-594 Oncolytic virus Advanced liver Started the Phase IIb trial in patients who (San Francisco) cancer failed prior therapy with Nexavar (11/7) KAEL-GemVax GV1001 Telomerase peptide Non-small-cell Interim Phase II data demonstrated an 80% Co. Ltd. (Seoul, lung cancer immune response rate (11/16) South Korea) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 12. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 12 OF 32 Company Product Description Indication Status (Date)# (Location) Ligand Melphalan Captisol-enabled Cancer Phase II data showed that it met the Pharmaceuticals propylene glycol- requirements for establishing bioequiva- Inc. (San Diego) free melphalan lence to Alkeran (11/3) MediGene AG EndoTAG-1 A composition of Hormone- Phase II trial started (11/9) (Martinsried, paclitaxel combined receptor- Germany) with neutral and positive, HER2- positive lipids negative breast cancer Peregrine Bavituximab A phosphatidylser- Locally Company reported a 23.2-month median Pharmaceuticals ine-targeting mono- advanced or overall survival from a single-arm Phase II Inc. (Tustin, Calif.) clonal antibody metastatic trial of bavituximab in combination with breast cancer carboplatin and paclitaxel, compared with a published study showing median OS of 16 months with carboplatin and paclitaxel alone (11/23) YM BioSciences Nimotuzumab Anti-EGFR antibody Recurrent dif- Phase II data showed it was well tolerated Inc. (Mississauga, fuse intrinsic when administered to pediatric patients Ont.) pontine glioma (11/1) CARDIOVASCULAR Aastrom Ixmyelocel-T A patient-specific Critical limb Phase II data showed patients in the treat- Biosciences Inc. multicellular therapy ischemia ment arm had a 62% reduction in risk rela- (Ann Arbor, Mich.) tive to placebo in the primary endpoint of time to first occurrence of treatment failure (11/15) Mesoblast Ltd. Revascor Stem cell treatment Congestive Phase II data showed it did not provoke any (Melbourne, heart failure adverse immune responses and there was Australia) a 78% reduction in the rate of serious cardi- ac events (11/16) Resverlogix RVX-208 A small molecule Coronary Started dosing in its Phase IIb study (11/3); Corp. (Calgary, designed to stimu- artery disease the study is fully enrolled with 176 subjects Alberta) late endogenous (11/29) ApoA-I production Sanofi SA (Paris) REGN727/ A fully human anti- Elevated low- Phase II data showed that after 12 weeks of and Regeneron SAR236553 body targeting pro- density lipo- treatment patients achieved mean LDL-C Pharmaceuticals protein convertase protein reductions ranging from about 30% to Inc. (Tarrytown, subtilisin/kexin cholesterol more than 65%, depending on the dosing N.Y.) type 9 regimen; the control arm had a mean reduction of 10% (11/11) Theravance Inc. TD-4208 Inhaled long-acting Chronic Phase IIa data showed it met the primary (South San muscarinic antago- obstructive endpoint, with both study doses demon- Francisco) nist pulmonary strating a statistically significant mean disease change from baseline in peak forced expi- ratory volume in one second compared to placebo (11/16) Trophos SA TRO40303 A mitochondria pore Cardiac isch- Treated the first patient in a Phase II study (Marseille, France) modulator emia-reperfu- (11/1) sion injury in acute myocar- dial infarction To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 13. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 13 OF 32 Company Product Description Indication Status (Date)# (Location) Verona Pharma RPL554 A mixed phosphodi- Chronic Demonstrated bronchodilator effects in a plc (London) esterase 3/4 inhibi- obstructive Phase IIa trial (11/11) tor pulmonary disease CENTRAL NERVOUS SYSTEM BioDelivery BEMA Bupre- Formulation of Opioid Started a confirmatory study, which will Sciences norphine/ buprenorphine and dependence select the final doses to be used in the piv- International Nalaxone naloxone using otal bioequivalence study compared to Inc. (Raleigh, N.C.) BDSI’s BioErodible Suboxone (11/11) MucoAdhesive drug delivery technology Clinuvel Scenesse Afamelanotide; pho- Erythropoietic Phase II data showed it appeared to be Pharmaceuticals toprotective drug protoporphyria effective in preventing severe pain result- Ltd. (Melbourne, ing from sun exposure (11/4) Australia) Cytokinetics Inc. CK-2017357 A fast skeletal mus- Amyotrophic Started a second cohort of an ongoing (South San cle troponin activator lateral Phase II trial (11/2); opened enrollment in its Francisco) sclerosis third Phase II trial (11/30) Knopp Dexpram- A small-molecule Amyotrophic Phase II data showed the highest dose of Biosciences LLC ipexole modulator of mito- lateral the drug reduced mortality by 68% in the (Pittsburgh) chondrial bioener- sclerosis second stage of the trial (11/22) getics NeurogesX Inc. NGX-1998 A topical liquid for- Postherpetic Phase II top-line data showed that the trial (San Mateo, Calif.) mulation of high- neuralgia met its primary endpoint of a percentage concentration change from baseline vs. placebo (11/9) capsaicin Rexahn Serdaxin A neuroprotective Major depres- Phase IIb data showed it did not demon- Pharmaceuticals and antidepressant sive disorder strate efficacy compared to placebo over Inc. (Rockville, candidate an eight-week treatment period (11/7) Md.) Supernus SPN-810 Molindone hydro- Impulsive Started a Phase IIb trial (11/29) Pharmaceuticals chloride aggression Inc. (Rockville, associated Md.) with attention deficit hyper- activity disorder INFECTION Alnylam ALN-RSV01 RNAi therapy Respiratory Completed enrollment in a Phase IIb trial Pharmaceuticals syncytial virus (11/30) Inc. (Cambridge, Mass.) Inviragen Inc. DENVax A dengue vaccine Dengue fever Started a Phase II trial (11/29) (Fort Collins, Colo.) based on an attenu- ated DEN-2 virus Medivir AB TMC435 An oral HCV prote- Chronic hepa- Phase IIb data showed that TMC435 once (Huddinge, ase inhibitor titis C virus daily in addition to pegylated interferon Sweden) and ribavirin produced superior rates of sustained virologic response compared to standard of care (11/3) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 14. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 14 OF 32 Company Product Description Indication Status (Date)# (Location) SEEK (London) FLU-v Vaccine Influenza The vaccine was well tolerated and effec- tive against all influenza strains in a Phase II challenge trial (11/8) Sinovac Biotech Vaccine Enterovirus 71 vac- Hand, foot and The vaccine had good immunogencity and Ltd. (Beijing) cine mouth disease a favorable safety profile with no adverse events in a Phase II trial (11/10) MISCELLANEOUS Alexion Soliris Eculizumab Atypical hemo- Pivotal Phase II data demonstrated that Pharmaceuticals lytic uremic ongoing treatment sustained the suppres- Inc. (Cheshire, syndrome sion of complement-mediated thrombotic Conn.) microangiopathy, maintained or further improved longer-term renal function and enhanced quality of life (11/11) Ampio Ampion A low molecular Nasal inflam- A double-blind study in 20 patients Pharmaceuticals fraction of commer- mation showed it was safe and effective for reduc- Inc. (Greenwood cial albumin ing nasal inflammation (11/11) Village, Colo.) FibroGen Inc. (San FG-4592 A hypoxia-inducible Chronic kidney Phase IIb data showed that 96% of patients Francisco) factor prolyl hydrox- disease had an increase in hemoglobin (Hb) of at ylase inhibitor least 1 g/dL, and 93% had an Hb response; treatment was well tolerated (11/16) Kythera ATX-101 Formulation of Submental fat Data from a long-term follow-up Phase IIa Biopharma- deoxycholate, an trial showed more than 90% of initial ceuticals Inc. endogenous com- responders sustained or improved their (Los Angeles) pound that pro- response (11/8) motes the natural breakdown of dietary fat ProFibrix BV Fibrocaps A dry powder topi- Liver resection Phase II data showed a 50% reduction in (Leiden, the cal fibrin sealant surgery mean time to hemostasis, the primary end- Netherlands) developed from a point, compared to active control (11/16) mixture of fibrino- gen and thrombin RegeneRx RGN-259 Sterile, preservative- Central corne- Phase II data showed it met statistical sig- Biopharma- free eye drop al fluorescein nificance after a challenge-controlled ceuticals Inc. staining adverse environment in the reduction from (Rockville, Md.) baseline compared to placebo (11/7) Repros Androxal Oral therapy that Low Phase II data showed that 25 mg of Therapeutics stimulates the pitu- testosterone Androxal showed statistically equivalent Inc. (The itary gland efficacy to topical Androgel (11/9) Woodlands, Texas) Ritter RP-G28 A drug designed to Lactose Last patient completed treatment in a Pharmaceuticals stimulate colonic intolerance Phase II study (11/18) Inc. (Los Angeles) growth Unigene Oral PTH Oral parathyroid Osteoporosis Phase II data showed it achieved the prima- Laboratories Inc. hormone analogue ry endpoint of increasing bone mineral (Boonton, N.J.) density at the lumbar spine (11/10) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 15. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 15 OF 32 Phase III Clinical Trials Update: November 2011 Company Product Description Indication Status (Date)# (Location) AUTOIMMUNE Genzyme Corp. Lemtrada Alemtuzumab Relapsing- Phase III data showed it met both of its (Cambridge, Mass.) remitting co-primary endpoints (11/15) multiple sclerosis NPS Natpara NPSP558; a bioengi- Hypopara- Phase III data showed that 53% of Pharmaceuticals neered replica of thyroidism Natpara-treated patients achieved the pri- Inc. (Bedminster, human parathyroid mary endpoint vs. 2% of placebo-treated N.J.) hormone patients (11/8) Nuron Biotech NU100 A recombinant Relapsing- Started a pivotal, Phase III trial (11/2) Inc. (Exton, Pa.) human interferon remitting multi- beta-1b ple sclerosis CANCER Amgen Inc. Xgeva Denosumab Bone metasta- Phase III data showed that Xgeva signifi- (Thousand Oaks, ses of prostate cantly prolonged bone metastasis-free Calif.) cancer survival by 4.2 months, delayed bone metastasis by 3.7 months and reduced symptoms of bone metastasis by 33% (11/17) Celgene Revlimid Lenalidomide Castrate- Company is terminating the pivotal Phase International resistant III MAINSAIL trial because the combina- Sarl (Boudry, prostate cancer tion of docetaxel and prednisone with Switzerland) Revlimid did not show a statistically sig- nificant improvement in overall survival compared to docetaxel and prednisone plus placebo (11/28) Exelixis Inc. Cabozantinib A dual inhibitor of End-stage cas- Company was unable to secure an SPA (South San MET and VEGFR trate resistant and plans to start a Phase III trial by the Francisco) prostate cancer end of the year with pain as the primary efficacy endpoint; a second Phase III trial to evaluate overall survival time will launch in 2012 (11/2) Medivation Inc. MDV3100 An oral androgen Prostate cancer A independent data monitoring commit- (San Francisco) receptor antagonist tee found Phase III interim data to be posi- tive and recommended the trial be stopped so the drug could be offered to men receiving placebo (11/4) Novocure Inc. TTF therapy Tumor treating Recurrent glio- Phase III data showed overall survival was (Anaheim, Calif.) fields therapy blastoma 9% vs. 7% and 8% vs. 1% for the TTF group multiforme compared to the standard of care at two and three years of follow-up, respectively (11/22) Talon Marqibo Vincristine sulfate Newly Enrolled the first patient in the Phase III Therapeutics Inc. liposomes injection diagnosed study of Marqibo (11/30) (San Mateo, Calif.) aggressive non-Hodgkin’s lymphoma To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 16. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 16 OF 32 Company Product Description Indication Status (Date)# (Location) CARDIOVASCULAR Almirall SA Aclidinium Fixed-dose combi- Chronic Began a Phase III trial (11/17) (Barcelona, Spain) bromide and nation via the obstructive pul- and Forest formoterol Genuari inhaler monary disease Laboratories Inc. fumarate (New York) Gilead Sciences Ranexa Ranolazine Chronic angina Started a Phase III trial (11/8) Inc. (Foster City, Calif.) Inspiration OBI-1 An intravenous Congenital Started patient enrollment in the seconf Biopharma- recombinant por- hemophilia A of two pivotal studies (11/28) ceuticals Inc. cine Factor VIII prod- (Laguna Niguel, uct Calif.) Omthera Epanova Triglyceride- High Completed enrollment in a pivotal, Phase Pharmaceuticals lowering therapy triglycerides III trial (11/30) Inc. (Bedminster, N.J.) Santarus Inc. (San Rhucin Recombinant Hereditary A retrospective analysis showed that Diego) human C1 inhibitor angioedema median time to primary endpoint of onset of symptom relief was 60 minutes and median time to minimal symptoms was 240 minutes (11/7) CENTRAL NERVOUS SYSTEM Alexza Adasuve Staccato loxapine Schizophrenia Phase III data showed a 40% or greater Pharmaceuticals or bipolar I decrease from baseline in the total PANSS- Inc. (Mountain disorder Excited Component score; another analy- View, Calif.) sis showed a statistically significant improvement over placebo using the Clinical Global Impression-Improvement scale (11/9) Alkermes plc Vivitrol Naltrexone Opioid Data showed sustained efficacy in those (Dublin) dependence receiving Vivitrol in combination with psychosocial treatment for 18 months (11/10) Nabi Biopharma- NicVAX Smoking cessation Smoking A second Phase III trial failed to meet its ceuticals Inc. vaccine cessation primary endpoint of abstinence from cig- (Rockville, Md.) arettes; the vaccine was well tolerated (11/8) Targacept Inc. TC-5214 Nicotinic channel Major depres- Failed to meet its primary endpoint of (Winston Salem, modulator to nor- sive disorder change on the Montgomery-Asberg N.C.) malize cholinergic Depression Rating Scale following eight tone weeks of treatment in a Phase III trial (11/9) Vivus Inc. Qnexa Phentermine/topira- Obesity Results from the 56-week EQUIP study in (Mountain View, mate 1 ,267 patients showed an average weight Calif.) loss of 14.4% of initial body weight, plus improvements in blood pressure, glucose, triglycerides and cholesterol (11/7) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.